NICE — Íàöèîíàëüíûé èíñòèòóò çäîðîâüÿ è êëèíè÷åñêîãî ñîâåðøåíñòâîâàíèÿ (êà÷åñòâà ìåäèöèíñêîé ïîìîùè) Âåëèêîáðèòàíèè (National Institute for Health and Clinical Excellence)
NK — åñòåñòâåííûå êëåòêè-êèëëåðû (natural killer)
NMDA — N-ìåòèë-D-àñïàðòàò (N-methyl-D-aspartate)
NMDA — N-ìåòèë-D-àñïàðòàòíûå ðåöåïòîðû
NNT — ÷èñëî áîëüíûõ, êîòîðûõ íåîáõîäèìî ïðîëå÷èòü, ÷òîáû äîñòè÷ü êàêîãî-òî óêàçàííîãî ýôôåêòà (Number Need to Treatment)
OR — îòíîøåíèå øàíñîâ (odds ratio)
paCO2 — ïàðöèàëüíîå äàâëåíèå óãëåêèñëîãî ãàçà â àðòåðèàëüíîé êðîâè
PALS — êóðñû ðàñøèðåííûõ ðåàíèìàöèîííûõ ìåðîïðèÿòèé â ïåäèàòðèè (Pediatric Advanced Life Support)
PANDAS — àóòîèììóííîå íåéðîïñèõèàòðè÷åñêîå ðàññòðîéñòâî â äåòñêîì âîçðàñòå, àññîöèèðîâàííîå ñî ñòðåïòîêîêêîâîé èíôåêöèåé (Pediatric autoimmune neuropsychiatric disorder associated with streptococcal infection)
 õîäå ïîäãîòîâêè ñòàòåé ïî äåòñêîé ãåìàòîëîãèè â ïåäèàòðèè äëÿ ïîëüçîâàòåëåé ñàéòà ÌåäÓíèâåð èñïîëüçîâàíû òðóäû ñëåäóþùèõ àâòîðîâ:
Christensen RD, Jopling J, Henry E, et al: The erythrocyte indices of neonates, defined using data from over 12,000 patients in a multihospital healthcare system, J Perinatol 28:24–28, 2008.
Deutsch VR, Toner A: Megakaryocyte development and platelet production, Br J Haematol 134:453–466, 2006.
Jopling J, Henry E, Wiedmeier SE, et al: Reference ranges for hematocrit and blood hemoglobin concentration during the neonatal period: data from a multihospital healthcare system, Pediatrics 123:e333–e337, 2009.
Julien E, Omar RE, Tavian M: Origin of the hematopoietic system in the human embryo, FEBS Lett 590:3987–4001, 2016.
Kaushansky K: Lineage-specific hematopoietic growth factors, N Engl J Med 354: 2034–2045, 2006.
Liang R, Ghaffari S: Advances in understanding the mechanisms of erythropoiesis in homeostasis and disease, Br J Haematol 174:661–673, 2016.
Liu A, Sola-Visner M: Neonatal and adult megakaryopoiesis, Curr Opin Hematol 18(5):330–337, 2011.
Sola-Visner MC, Christensen RD, Hutson AD, et al: Megakaryocyte size and concentration in the bone marrow of thrombocytopenic and nonthrombocytopenic neonates, Pediatr Res 61:479–484, 2007.
Spangrude GJ, Perry SS, Slayton WB: Early stages of hematopoietic differentiation, Ann NY Acad Sci 996:186–194, 2003.
Vats A, Bielby RC, Tolley NS, et al: Stem cells, Lancet 366:592–602, 2005.
Wang Y, Hayes V, Jarocha D, et al: Comparative analysis of human ex vivo–generated platelets vs megakaryocyte-generated platelets in mice: a cautionary tale, Blood 125(23):3627–3636, 2015.
Wiedmeier SE, Henry E, Sola-Visner MC, et al: Platelet reference ranges for neonates, defined using data from over 47,000 patients in a multihospital healthcare system, J Perinatol 29:130–136, 2009.
Yoshimoto M, Yoder MC: Developmental biology: birth of the blood cell, Nature 457:801–813, 2009.
Powell DJ, Achebe MO: Anemia for the primary care physician, Prim Care 43(4):527–542, 2016.
Wang M: Iron deficiency and other types of anemia in infants and children, Am Fam Physician 93(4):270–278, 2016.
Crazzolara R, Kropshofer G, Haas OA, et al: Reduced-intensity conditioning and stem cell transplantation in infants with Diamond-Blackfan anemia, Haematologica 102(3):e73–e75, 2017.
Dietz AC, Mehta PA, Vlachos A, et al: Current knowledge and priorities for future research in late effects after hematopoietic cell transplantation (HCT) for inherited bone marrow failure syndromes (IBMFS): consensus statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric HCT, Biol Blood Marrow Transplant 23(5):726–735, 2017.
Ikeda F, Yoshida K, Toki T, et al: Exome sequencing identified RPS15A as a novel causative gene for Diamond-Blackfan anemia, Haematologica 102(3):e93–e96, 2017.
Lahoti A, Harris YT, Speiser PW, et al: Endocrine dysfunction in Diamond-Blackfan anemia (DBA): a report from the DBA Registry (DBAR), Pediatr Blood Cancer 63:306–312, 2016.
Ludwig LS, Gazda HT, Eng JC, et al: Altered translation of GATA1 in Diamond-Blackfan anemia, Nat Med 20:748–753, 2014.
Narla A, Davis NL, Lavasseur C, et al: Erythrocyte adenosine deaminase levels are elevated in Diamond-Blackfan anemia but not in the 5q-syndrome, Am J Hematol 91:E501–E502, 2016.
Narla A, Yuan D, Kazerounian S, et al: A novel pathogenic mutation in RPL11 identified in a patient diagnosed with Diamond-Blackfan anemia as a young adult, Blood Cells Mol Dis 61:46–47, 2016.
Parrella S, Aspesi A, Quarello P, et al: Loss of GATA-1 full length as a cause of Diamond-Blackfan anemia phenotype, Pediatr Blood Cancer 61:1319–1321, 2014.
Vlachos A, Blanc L, Lipton JM: Diamond-Blackfan anemia: a model for the translational approach to understanding human disease, Expert Rev Hematol 7:359–372, 2014.
Alter BP: Pearson syndrome in a Diamond-Blackfan anemia cohort, Blood 124:312–313, 2014.
Gagne KE, Ghazvinian R, Yuan D, et al: Pearson marrow pancreas syndrome in patients suspected to have Diamond-Blackfan anemia, Blood 124:437–440, 2014.
Park J, Ryu H, Jang W, et al: Novel 5.712 kb mitochondrial DNA deletion in a patient with Pearson syndrome: a case report, Mol Med Rep 11:3741–3745, 2015.
Sato T, Muroya K, Hanakawa J, et al: Clinical manifestations and enzymatic activities of mitochondrial respiratory chain complexes in Pearson marrow-pancreas syndrome with 3-methylglutaconic aciduria: a case report and literature review, Eur J Pediatr 174:1593–1602, 2015.
Shimamura A, Alter BP: Pathophysiology and management of inherited bone marrow failure syndromes, Blood Rev 24:101–122, 2010.
Aoki K, Ono Y, Tabata S, et al: Successful treatment of anti-erythropoietin antibody–mediated pure red cell aplasia with low-dose prednisolone, Int J Hematol 97:272–274, 2013.
Crabol Y, Terrier B, Rozenberg F, et al; Groupe d’experts de l’Assistance PubliqueHopitaux: Intravenous immunoglobulin therapy for pure red cell aplasia related to human parvovirus B19 infection: a retrospective study of 10 patients and review of the literature, Clin Infect Dis 56:968–977, 2013.
Karabulut A, Gok S, Kocyigit A: Non–immune hydrops fetalis without anemia due to parvovirus B19, Int J Gynaecol Obstet 124:82, 2014.
MacDougall IC, Casadevall N, Locatelli F, et al: Incidence of erythropoietin antibody–mediated pure red cell aplasia: the Prospective Immunogenicity Surveillance Registry (PRIMS), Nephrol Dial Transplant 30:451–460, 2015.
Makhlouf MM, Elwakil SG, Ibrahim NS: Molecular and serological assessment of parvovirus B-19 infection in Egyptian children with sickle cell disease, J Microbiol Immunol Infect 2015 Dec 1. pii: S1684-1182(15), 00914-7.
Ruiz Gutierrez L, Albarran F, Moruno H, Cuende E: Parvovirus B19 chronic monoarthritis in a patient with common variable immunodeficiency, Reumatol Clin 11:58–59, 2015.
Van den Akker M, Dror Y, Odame I: Transient erythroblastopenia of childhood is an underdiagnosed and self-limiting disease, Acta Paediatr 103:e288–e294, 2014.
Welcker S, Heckmann M: Non-immune hydrops fetalis due to parvovirus B19 infection in 2 extremely preterm infants: perinatal management and long-term neurodevelopmental outcome, Z Geburtshilfe Neonatol 219:144–147, 2015.
Xu LH, Fang JP, Weng WJ, et al: Pure red cell aplasia associated with cytomegalovirus and Epstein-barr virus infection in seven cases of Chinese children, Hematology 18:56–59, 2013.
Camaschella C: Iron and hepcidin: a story of recycling and balance, Hematology 2013:1–8, 2013.
Fraenkel PG: Understanding anemia of chronic disease, Hematology 2015:14–18, 2015.
Gangat N, Wolanskyj AP: Anemia of chronic disease, Semin Hematol 50(3):232–238, 2013.
Poggiali E, Migone De Amicis M, Motta I: Anemia of chronic disease: a unique defect of iron recycling for many different chronic diseases, Eur J Intern Med 25:12–17, 2014.
Turgeon O’Brien H, Blanchet R, Gagn? D, et al: Using soluble transferrin receptor and taking inflammation into account when defining serum ferritin cutoffs improved the diagnosis of iron deficiency in a group of Canadian preschool inuit children from Nunavik, Anemia 2016:6430214, 2016.
Atkinson MA, Martz K, Warady BA, Neu AM: Risk for anemia in pediatric chronic kidney disease patients: a report of NAPRTCS, Pediatr Nephrol 25:1699–1706, 2010.
Atkinson MA, Kim JY, Roy CN, et al: Hepcidin and risk of anemia in CKD: a cross-sectional and longitudinal analysis in the CKiD cohort, Pediatr Nephrol 30:635–643, 2015.
Girelli D, Ugolini S, Busti F, et al: Modern iron replacement therapy: clinical and pathophysiological insights, Int J Hematol 107(1):16–30, 2018.
Goldstein AL, Morris D, Warady BA: Comparison of the safety and efficacy of 3 iron sucrose iron maintenance regimens in children, adolescents, and young adults with CKD: a randomized controlled trial, Am J Kidney Dis 61:588–597, 2013.
Warady BA, Neu AM, Schaefer F: Optimal care of the infant, child, and adolescent on dialysis: 2014 update, Am J Kidney Dis 64:128–142, 2014.
Warady BA, Silverstein DM: Management of anemia with erythropoietic-stimulating agents in children with chronic kidney disease, Pediatr Nephrol 29:1493–1505, 2014.
Babbs C, Roberts NA, Sanchez-Pulido L, et al: Homozygous mutations in a predicted endonuclease are a novel cause of congenital dyserythropoietic anemia type I, Haematologica 98:1383–1387, 2013.
Bianchi P, Schwarz K, Hogel J, et al: Analysis of a cohort of 101 CDAII patients: description of 24 new molecular variants and genotype-phenotype correlations, Br J Haematol 175:696–704, 2016.
Gambale A, Iolascon A, Andolfo I, Russo R: Diagnosis and management of congenital dyserythropoietic anemias, Expert Rev Hematol 9:283–296, 2016.
Iolascon A, Heimpel H, Wahlin A, Tamary H: Congenital dyserythropoietic anemias: molecular insights and diagnostic approach, Blood 122:2162–2166, 2013.
Liljeholm M, Irvine AF, Vikberg AL, et al: Congenital dyserythropoietic anemia type III (CDA III) is caused by a mutation in kinesin family member, KIF23, Blood 121:4791–4799, 2013.
Russo R, Gambale A, Langella C, et al: Retrospective cohort study of 205 cases with congenital dyserythropoietic anemia type II: definition of clinical and molecular spectrum and identification of new diagnostic scores, Am J Hematol 89:E169–E175, 2014.
Shalev H, Al-Athamen K, Levi I, et al: Morbidity and mortality of adult patients with congenital dyserythropoietic anemia type I, Eur J Haematol 98:13–18, 2017.
Anemia of prematurity. In Malcolm W, editor: Beyond the NICU: comprehensive care of the high-risk infant, New York, 2015, McGraw-Hill Education, pp 202–210.
Banerjee J, Aladangady N: Biomarkers to decide red blood cell transfusion in newborn infants, Transfusion 54:2574–2582, 2014.
Berglund SK, Westrup B, Domell?f M: Iron supplementation until 6 month protects marginally low-birth-weight infants from iron deficiency during their first year of life, JPGN 60(3):390–395, 2015.
Katheria AC, Truong G, Cousins L, et al: Umbilical cord milking versus delayed cord clamping in preterm infants, Pediatrics 136:61–69, 2015.
Ohls RK, Christensen RD, Kamath-Rayne BD, et al: A randomized, masked, placebocontrolled study of darbepoetin alfa in preterm infants, Pediatrics 132:e119–e127, 2013.
Ohls RK, Kamath-Rayne BD, Christensen RD, et al: Cognitive outcomes of preterm infants randomized to darbepoetin, erythropoietin, or placebo, Pediatrics 133: 1023–1030, 2014.
Whyte RK, Jefferies AL, French Canadian Paediatric Society, Newborn C: Red blood cell transfusion in newborn infants, Paediatr Child Health 19:213–222, 2014.
Zhang Y, Jin L, Liu JM, et al: Maternal hemoglobin concentration during gestation and risk of anemia in infancy: secondary analysis of a randomized controlled trial, J Pediatr 175:106–110 e2, 2016.
Alpers DH: Absorption and blood/cellular transport of folate and cobalamin: pharmacokinetic and physiological considerations, Biochimie 126:52–56, 2016.
D’Aco KE, Bearden D, Watkins D, et al: Severe 5,10-methylenetetrahydrofolate reductase deficiency and two MTHFR variants in an adolescent with progressive myoclonic epilepsy, Pediatr Neurol 51:266–270, 2014.
Ruiz-Mercado M, Vargas MT, de Soto IP, et al: Methionine synthase reductase deficiency (CblE): a report of two patients and a novel mutation, Hematology 21:193–197, 2016.
Zhao R, Aluri S, Goldman ID: The proton-coupled folate transporter (PCFT-SLC46A1) and the syndrome of systemic and cerebral folate deficiency of infancy: hereditary folate malabsorption, Mol Aspects Med 53:57–72, 2017.
Bahadir A, Reis PG, Erduran E: Oral vitamin B12 treatment is effective for children with nutritional vitamin B12 deficiency, J Paediatr Child Health 50:721–725, 2014.
Ekici F, Tekbas G, Hattapoglu S, et al: Brain MRI and MR spectroscopy findings in children with nutritional vitamin B12 deficiency, Clin Neuroradiol 26:215–220, 2016.
Gherasim C, Lofgren M, Banerjee R: Navigating the B(12) road: assimilation, delivery, and disorders of cobalamin, J Biol Chem 288:13186–13193, 2013.
Jain R, Singh A, Mittal M, Talukdar B: Vitamin B12 deficiency in children: a treatable cause of neurodevelopmental delay, J Child Neurol 30:641–643, 2015.
Krzemien G, Turczyn A, Szmigielska A, Roszkowska-Blaim M: Vitamin B12 deficiency in children (Imerslund-Grasbeck syndrome in two pairs of siblings), Dev Period Med 19:351–355, 2015.
Manios Y, Moschonis G, Dekkers R, et al: Vitamin B2, vitamin B12 and total homocysteine status in children and their associations with dietary intake of B-vitamins from different food groups: the Healthy Growth Study, Eur J Nutr 1:321–331, 2017.
Montgomery E, Sayer JA, Baines LA, et al: Novel compound heterozygous mutations in AMN cause Imerslund-Grasbeck syndrome in two half-sisters: a case report, BMC Med Genet 16:35, 2015.
Stabler SP: Clinical practice. Vitamin B12 deficiency, N Engl J Med 368:149–160, 2013.
Sturm AC, Baack EC, Armstrong MB, et al: Hereditary intrinsic factor deficiency in Chaldeans, JIMD Rep 7:13–18, 2013.
Trakadis YJ, Alfares A, Bodamer OA, et al: Update on transcobalamin deficiency: clinical presentation, treatment and outcome, J Inherit Metab Dis 37:461–473, 2014.
Nyhan WL, Gangoiti JA: Hereditary orotic aciduria and the excretion of orotidine, Neuropediatrics 47:408–409, 2016.
Setoodeh A, Haghighi A, Saleh-Gohari N, et al: Identification of a SLC19A2 nonsense mutation in Persian families with thiamine-responsive megaloblastic anemia, Gene 519:295–297, 2013.
Abdullah K, Birken CS, Maguire JL, et al: Re-evaluation of serum ferritin cut-off values for the diagnosis of iron deficiency in children aged 12-36 months, J Pediatr 188:287–290, 2017.
American Academy of Pediatrics: Bright Futures: recommendations for preventive pediatric healthcare. https://brightfutures.aap.org/Pages/default.aspx
Andrews NC, Ullrich CK, Fleming MD, et al: Disorders of iron metabolism and sideroblastic anemia. In Orkin SH, Look AT, Nathan DG, editors: Nathan and Oski’s hematology of infancy and childhood, ed 7, Philadelphia, 2009, Saunders.
Ashish KC, Rana N, Malqvist M, et al: Effects of delayed umbilical cord clamping vs early clamping on anemia in infants at 8 and 12 months, JAMA Pediatr 171(3):264–270, 2017.
Baker RD, Greer FR, Committee on Nutrition: Clinical report: diagnosis and prevention of iron deficiency and iron deficiency anemia in infants and young children (0-3 years of age), Pediatrics 126:1040–1050, 2010.
Berglund SK, Westrup B, Domell?f M: Iron supplementation until 6 months protects marginally low-birth-weight infants from iron deficiency during their first year of life, J Pediatr Gastroenterol Nutr 60(3):390–395, 2015.
Berglund SK, Westrup B, H?ggl?f B, et al: Effects of iron supplementation of LBW infants on cognition and behavior at 3 years, Pediatrics 131(1):47–55, 2013.
Buchanan GR: Paucity of clinical trials in iron deficiency: lessons learned from study of VLBW infants, Pediatrics 131:e582–e584, 2013.
Clark KM, Li M, Zhu B, et al: Breastfeeding, mixed, or formula feeding at 9 months of age and the prevalence of iron deficiency and iron deficiency anemia in two cohorts of infants in China, J Pediatr 181:56–61, 2017.
Committee Opinion No 684: Delayed umbilical cord clamping after birth, Obstet Gynecol 129(1):e5–e10, 2017.
Congdon EL, Westerlund A, Algarin C, et al: Iron deficiency in infancy is associated with altered neural correlates of recognition memory at 10 years, J Pediatr 160:1027–1033, 2012.
DeLoughery TG: Microcytic anemia, N Engl J Med 371:1324–1331, 2014.
Falkingham M, Abdelhamid A, Curtis P, et al: The effects of oral iron supplementation on cognition in older children and adults: a systematic review and meta-analysis, Nutr J 9:4, 2010.
Ferrara M, Capozzi L, Russo R, et al: Reliability of red blood cell indices and formulas to discriminate between beta thalassemia trait and iron deficiency in children, Hematology 15(2):112–115, 2010.
Fleming MD, et al: Disorders of iron and copper metabolism, the sideroblastic anemias, and lead toxicity. In Orkin SH, Fisher DE, Ginsberg D, editors: Nathan and Oski’s hematology and oncology of infancy and childhood, ed 8, Philadelphia, 2005, Saunders.
Kiss JE, Brambilla D, Glynn Sa, et al: Oral iron supplementation after blood donation: a randomized clinical trial, JAMA 313(6):575–583, 2015.
Khuong-Quang DA, Schwartzentruber J, Westerman M, et al: Iron refractory iron deficiency anemia: presentation with hyperferritinemia and response to oral iron therapy, Pediatrics 131:e620–e625, 2013.
Litton E, Xiao J, Ho KM: Safety and efficacy of intravenous iron therapy in reducing requirement for allogeneic blood transfusion: systematic review and meta-analysis of randomized clinical trials, BMJ 347:f4822, 2013.
Lopez A, Cacoub P, Macdougall C, Peyrin-Biroulet L: Iron deficiency anaemia, Lancet 387:907–916, 2016.
Lukowski AF, Koss M, Burden MJ: Iron deficiency in infancy and neurocognitive functioning at 19 years: evidence of long-term deficits in executive function and recognition memory, Nutr Neurosci 13:54–70, 2010.
Maguire JL, Salehi L, Birken CS, et al: Association between total duration of breastfeeding and iron deficiency, Pediatrics 131:e1530–e1537, 2013.
McDonald SJ, Middleton P, Dowswell T, et al: Effect of timing of umbilical cord clamping of term infants on maternal and neonatal outcomes, Cochrane Database Syst Rev (7):CD004074, 2013.
McDonagh MS, Blazina I, Dana T, et al: Screening and routine supplementation for iron deficiency anemia: a systematic review, Pediatrics 135(4):723–733, 2015.
The Medical Letter: Triferic for iron replacement, Med Lett 59(1517):55–56, 2017.
The Medical Letter: Ferric carboxymaltose (Injectafer) for iron deficiency anemia, Med Lett 55:99–100, 2013.
Miller JL: Iron deficiency anemia: a common and curable disease, Cold Spring Harb Perspect Med 3(7):a011866, 2013.
National Institutes of Health, Office of Dietary Supplements: Dietary supplement fact sheet: iron. https://ods.od.nih.gov/factsheets/Iron-HealthProfessional/.
Pasricha SR, Atkinson SH, Armitage AE, et al: Expression of the iron hormone hepcidin distinguishes different types of anemia in African children, Sci Transl Med 6(255):235re3, 2014.
Powers JM, Buchanan GR: Potential for improved screening, diagnosis and treatment for iron deficiency and iron deficiency anemia in young children, J Pediatr 188:8–10, 2017.
Powers JM, Buchanan GR: Diagnosis and management of iron deficiency anemia, Hematol Oncol Clin North Am 28(4):729–745, 2014.
Powers JM, Buchanan GR, Adix L, et al: Effect of low-dose ferrous sulfate vs iron polysaccharide complex on hemoglobin concentration in young children with nutritional iron-deficiency anemia, JAMA 317(22):2297–2304, 2017.
Powers JM, Shamoun M, McCavit TL, et al: Intravenous ferric carboxymaltose in children with iron deficiency anemia who respond poorly to oral iron, J Pediatr 180:212–216, 2016.
Siu AL: Screening for iron deficiency anemia in young children: USPSTF recommendation statement, Pediatrics 136(4):746–752, 2015.
Soofii S, Cousens S, Iqbal SP, et al: Effect of provision of daily zinc and iron with several micronutrients on growth and morbidity among young children in Pakistan: a cluster-randomised trial, Lancet 382:29–40, 2013.
Thompson J, Biggs BA, Pasricha SR: Effects of daily iron supplementation in 2- to 5-year-old children: systematic review and meta-analysis, Pediatrics 131(4):739–753, 2013.
Torsvik IK, Markestad T, Ueland PM, et al: Evaluating iron status and the risk of anemia in young using erythrocyte parameters, Pediatr Res 73(2):214–220, 2013.
Zehetner AA, Orr N, Buckmast A, et al: Iron supplementation for the treatment of breath-holding spells in children, Cochrane Database Syst Rev 7:1–25, 2010.
Arsenault V, Mailloux C, Bonnefoy A, et al: Iron-refractory iron deficiency anemia may not lead to neurocognitive dysfunction: a case report, Pediatrics 138(1):e1–e4, 2016.
Avni T, Bieber A, Grossman A, et al: The safety of intravenous iron preparations: systematic review and meta-analysis, Mayo Clin Proc 90(1):12–23, 2015.
Camaschella C: New insights into iron deficiency and iron deficiency anemia, Blood Rev 31(4):225–233, 2017.
De Falco L, Sanchez M, Silvestri L, et al: Iron refractory iron deficiency anemia, Haematologica 98(6):845–853, 2013.
Donker AE, Raymakers RA, Vlasveld LT, et al: Practice guidelines for the diagnosis and management of microcytic anemias due to genetic disorders of iron metabolism or heme synthesis, Blood 123(25):3873–3886, quiz 4005, 2014.
Heeney MM, Finberg KE: Iron-refractory iron deficiency anemia (IRIDA), Hematol Oncol Clin North Am 28(4):637–652, v, 2014.
Hershko C, Camaschella C: How I treat unexplained refractory iron deficiency anemia, Blood 123(3):326–333, 2014.
Sangkhae V, Nemeth E: Regulation of the iron homeostatic hormone hepcidin, Adv Nutr 8(1):126–136, 2017.
Wang CY, Meynard D, Lin HY: The role of TMPRSS6/matriptase-2 in iron regulation and anemia, Frontiers Pharmacol 5:114, 2014.
Andrews NC, Ullrich CK, Fleming MD: Disorders of iron metabolism and sideroblastic anemia. In Orkin SH, Look AT, Nathan DG, et al, editors: Nathan and Oski’s hematology of infancy and childhood, ed 7, Philadelphia, 2009, Saunders.
Beaumont-Epinette MP, et al: Hereditary hypotransferrinemia can lead to elevated transferrin saturation and, when associated to HFE or HAMP mutations, to iron overload, Blood Cells Mol Dis 54(2):151–154, 2015.
Camaschella C: How I manage patients with atypical microcytic anaemia, Br J Haematol 160:12–24, 2012.
Dembinski K, Gargasz AE, Dabrow S, et al: Three distinct cases of copper deficiency in hospitalized pediatric patients, Clin Pediatr 51:759–762, 2012.
Donker AE, et al: Practice guidelines for the diagnosis and management of microcytic anemias due to genetic disorders of iron metabolism or heme synthesis, Blood 123(25):3873–3886, 2014.
Fleming MD: Disorders of iron and copper metabolism, the sideroblastic anemias, and lead toxicity. In Orkin SH, Fisher DE, Ginsberg D, et al, editors: Nathan and Oski’s hematology and oncology of infancy and childhood, ed 8, Philadelphia, 2005, Saunders.
Miyajima H: Aceruloplasminemia, 2003 [updated 2015]. In Pagon RA, Adam MP, Ardinger HH, et al, editors: GeneReviews [internet], Seattle, 1993–2017, University of Washington.
Andolfo I, Russo R, Gambale A, Iolascon A: New insights on hereditary erythrocyte membrane defects, Haematologica 101:1284–1294, 2016.
Bolton-Maggs PHB, Langer JC, Iolascon A, et al: Guidelines for the diagnosis and management of hereditary spherocytosis—2011 update, Br J Haematol 156:37–49, 2011.
Christensen RD, Yaish HM, Gallagher PG: A pediatrician’s practical guide to diagnosing and treating hereditary spherocytosis in neonates, Pediatrics 135:1107–1114, 2015.
Englum BR, Rothman J, Leonard S, et al: Hematologic outcomes after total splenectomy and partial splenectomy for congenital hemolytic anemia, J Pediatr Surg 51:122–127, 2016.
Gallagher PG: Abnormalities of the erythrocyte membrane, Pediatr Clin North Am 60:1349–1362, 2013.
Iolascon A, Andolfo I, Barcellini W, et al: Recommendations for splenectomy in hereditary hemolytic anemias, Haematologica 102:1304–1313, 2017.
Narla J, Mohandas N: Red cell membrane disorders, Int J Lab Hematol 39(S1):47–52, 2017.
Pincez T, Guitton C, Gauthier F, et al: Long-term follow-up of subtotal splenectomy for hereditary spherocytosis: a single-center study, Blood 127:1616–1618, 2016.
Rice HE: Understanding splenectomy for children with hereditary spherocytosis, Pediatr Blood Cancer 63:1700–1701, 2016.
Andolfo I, Russo R, Gambale A, Iolascon A: New insights on hereditary erythrocyte membrane defects, Haematologica 101:1284–1294, 2016.
Gallagher PG: Abnormalities of the erythrocyte membrane, Pediatr Clin North Am 60:1349–1362, 2013.
Gallagher PG: Hereditary elliptocytosis: spectrin and protein 4.1r, Semin Hematol 41:142–164, 2004.
Iolascon A, Andolfo I, Barcellini W, et al: Recommendations for splenectomy in hereditary hemolytic anemias, Haematologica 102:1304–1313, 2017.
Narla J, Mohandas N: Red cell membrane disorders, Int J Lab Hematol 39(S1):47–52, 2017.
Alper SL: Genetic diseases of PIEZO1 and PIEZO2 dysfunction, Curr Top Membr 79:97–134, 2017.
Andolfo I, Russo R, Gambale A, Iolascon A: New insights on hereditary erythrocyte membrane defects, Haematologica 101:1284–1294, 2016.
Gallagher PG: Diagnosis and management of rare congenital nonimmune hemolytic disease, Hematology Am Soc Hematol Educ Program 2015:392–399, 2015.
Glogowska E, Schneider ER, Maksimova Y, et al: Novel mechanisms of PIEZO1 dysfunction in hereditary xerocytosis, Blood 130(16):1845–1856, 2017.
Glogowska E, Lezon-Geyda K, Maksimova Y, et al: Mutations in the gardos channel (KCNN4) are associated with hereditary xerocytosis, Blood 126:1281–1284, 2015.
Glogowska E, Gallagher PG: Disorders of erythrocyte volume homeostasis, Int J Lab Hematol 37(S1):85–91, 2015.
Iolascon A, Andolfo I, Barcellini W, et al: Recommendations for splenectomy in hereditary hemolytic anemias, Haematologica 102:1304–1313, 2017.
Narla J, Mohandas N: Red cell membrane disorders, Int J Lab Hematol 39(S1):47–52, 2017.
Adjobo-Hermans MJ, Cluitmans JC, Bosman GJ: Neuroacanthocytosis: observations, theories and perspectives on the origin and significance of acanthocytes, Tremor Other Hyperkinet Mov (N Y) 5:328, 2015.
Denomme GA: Kell and kx blood group systems, Immunohematology 31:14–19, 2015.
DeZern AE, Brodsky RA: Paroxysmal nocturnal hemoglobinuria: a complementmediated hemolytic anemia, Hematol Oncol Clin North Am 29:479–494, 2015.
Hill A, DeZern AE, Kinoshita T, Brodsky RA: Paroxysmal nocturnal haemoglobinuria, Nat Rev Dis Primers 3:17028, 2017.
Kelly RJ, Hochsmann B, Szer J, et al: Eculizumab in pregnant patients with paroxysmal nocturnal hemoglobinuria, N Engl J Med 373(11):1032–1038, 2015.
McNamara LA, Topaz N, Wang X, et al: High risk for invasive meningococcal disease among patients receiving eculizumab (Soliris) despite receipt of meningococcal vaccine, MMWR Morb Mortal Wkly Rep 66(27):734–736, 2017.
Mercuri A, Farruggia P, Timeus F, et al: A retrospective study of paroxysmal nocturnal hemoglobinuria in pediatric and adolescent patients, Blood Cells Mol Dis 64:45–50, 2017.
Parker CJ: Update on the diagnosis and management of paroxysmal nocturnal hemoglobinuria, Hematology Am Soc Hematol Educ Program 1:208–216, 2016.
Risitano AM, Marotta S: Therapeutic complement inhibition in complement-mediated hemolytic anemias: past, present and future, Semin Immunol 28:223–240, 2016.
Reiss UM, Schwartz J, Sakamoto KM, et al: Efficacy and safety of eculizumab in children and adolescents with paroxysmal nocturnal hemoglobinuria, Pediatr Blood Cancer 61:1544–1550, 2014.
Shen W, Clemente MJ, Hosono N, et al: Deep sequencing reveals stepwise mutation acquisition in paroxysmal nocturnal hemoglobinuria, J Clin Invest 124:4529–4538, 2014.
Walker RH: Untangling the thorns: advances in the neuroacanthocytosis syndromes, J Mov Disord 8:41–54, 2015.
Abboud MR, Yim E, Musallam KM, et al: Discontinuing prophylactic transfusions increases the risk of silent brain infarction in children with sickle cell disease: data from STOP II, Blood 118(4):894–898, 2011.
Ataga KI, Kutlar A, Kanter J, et al: Crizanlizumab for the prevention of pain crises in sickle cell disease, N Engl J Med 376(5):429–438, 2017.
Ballas SK, Bauserman RL, McCarthy WF, et al: Hydroxyurea and acute painful crises in sickle cell anemia: effects on hospital length of stay and opioid utilization during hospitalization, outpatient acute care contacts, and at home, J Pain Symptom Manage 40(6):870–882, 2010.
Baskin MN, Goh XL, Heeney MM, et al: Bacteremia risk and outpatient management of febrile patients with sickle cell disease, Pediatrics 131(6):1035–1041, 2013.
Belmont AP, Nossair F, Brambilla D, et al: Safety of deep sedation in young children with sickle cell disease: a retrospective cohort study, J Pediatr 166:1226–1232, 2015.
Berger E, Saunders N, Wang L, et al: Sickle cell disease in children, Arch Pediatr Adolesc Med 163:251255, 2009.
Beyer JE, Simmons LE, Woods GM, et al: A chronology of pain and comfort in children with sickle cell disease, Arch Pediatr Adolesc Med 153(9):913–920, 1999.
Bolanos-Meade J, Fuchs EJ, Luznik L, et al: HLA-haploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease, Blood 120(22):4285–4291, 2012.
Brandow AM, Zappia KJ, Stucky CL: Sickle cell disease: a natural model of acute and chronic pain, Pain 158:S79–S84, 2017.
Brousse V, Elie C, Benkerrou M, et al: Acute splenic sequestration crisis in sickle cell disease: cohort study of 190 paediatric patients, Br J Haematol 156(5):643–648, 2012.
Brousseau DC, McCarver DG, Drendel AL, et al: The effect of CYP2d6 polymorphisms on the response to pain treatment for pediatric sickle cell pain crisis, J Pediatr 150(6):623–626, 2007.
Brousseau DC, Panepinto JA, Nimmer M, et al: The number of people with sickle-cell disease in the United States: national and state estimates, Am J Hematol 85(1):77–78, 2010.
Buchanan ID, Woodward M, Reed GW: Opioid selection during sickle cell pain crisis and its impact on the development of acute chest syndrome, Pediatr Blood Cancer 45(5):716–724, 2005.
Dallas MH, Triplett B, Shook DR, et al: Long-term outcome and evaluation of organ function in pediatric patients undergoing haploidentical and matched related hematopoietic cell transplantation for sickle cell disease, Biol Blood Marrow Transplant 19(5):820–830, 2013.
Davis BA, Allard S, Qureshi A, et al: British committee for standards in haematology: guidelines on red cell transfusion in sickle cell disease. Part I. Principles and laboratory aspects, Br J Haematol 176:179–191, 2017.
DeBaun MR, Gordon M, McKinstry MJ, et al: Controlled trial of transfusions for silent cerebral infarcts in sickle cell anemia, N Engl J Med 371:699–710, 2014.
DeBaun MR, Sarnaik SA, Rodeghier MJ, et al: Associated risk factors for silent cerebral infarcts in sickle cell anemia: low baseline hemoglobin, gender and relative high systolic blood pressure, Blood 119:3684–3690, 2012.
DeBaun MR, Struck RC: The intersection between asthma and acute chest syndrome in children with sickle-cell anaemia, Lancet 387:2545–2552, 2016.
Dever DP, Bal RO, Reinisch A, et al: CRISPR/cas9 ?-globin gene targeting in human haematopoietic stem cells, Nature 539(7629):384–389, 2016.
Dowling MM, Quinn CT, Plumb P, et al: Acute silent cerebral ischemia and infarction during acute anemia in children with and without sickle cell disease, Blood 120:3891–3897, 2012.
Ellison AM, Ota KV, McGowan KL, et al: Pneumococcal bacteremia in a vaccinated pediatric sickle cell disease population, Pediatr Infect Dis J 5:534–536, 2012.
Field JJ, Austin PF, An P, et al: Enuresis is a common and persistent problem among children and young adults with sickle cell anemia, Urology 72:81–84, 2008.
Frenette PS, Atweh GF: Sickle cell disease: old discoveries, new concepts, and future promise, J Clin Invest 117:850–858, 2007. Gillis VL, Senthinathan A, Dzingina M, et al: Management of an acute painful sickle cell episode in hospital: summary of NICE guidance, BMJ 344:e4063, 2012.
Gladwin MT: Cardiovascular complications and risk of death in sickle-cell disease, Lancet 387:2565–2572, 2016.
Gluckman E, Cappelli B, Bernaudin F, et al: The pediatric working party of the European society for blood and marrow transplantation, and the center for international blood and marrow transplant research. Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation, Blood 129:1548–1556, 2017.
Halasa NB, Shankar SM, Talbot TR, et al: Incidence of invasive pneumococcal disease among individuals with sickle cell disease before and after the introduction of the pneumococcal conjugate vaccine, Clin Infect Dis 44:1428–1433, 2007.
Hines PC, McKnight TP, Seto W, et al: Central nervous system events in children with sickle cell disease presenting acutely with headache, J Pediatr 159:472–478, 2011.
Hsieh MM, Fithugh CD, Weitzel P, et al: Nonmyeloablative HLA-matched sibling allogeneic hematopoietic stem cell transplantation for severe sickle cell phenotype, JAMA 312:48–56, 2014.
Hoppe C: Newborn screening for hemoglobin disorders, Hemoglobin 35(5/6):556–564, 2011.
Huang X, Wang Y, Yan W, et al: Production of gene-corrected adult beta globin protein in human erythrocytes differentiated from patient IPSCs after genome editing of the sickle point mutation, Stem Cells 33(5):1470–1479, 2015.
Hulbert ML, Scothorn DJ, Panepinto JA, et al: Exchange blood transfusion compared with simple transfusion for first overt stroke is associated with a lower risk of subsequent stroke: a retrospective cohort study of 137 children with sickle cell anemia, J Pediatr 149(5):710–712, 2006.
Jain R, Sawhney S, Rizvi SG: Acute bone crises in sickle cell disease: the T1 fat-saturated sequence in differentiation of acute bone infarcts from acute osteomyelitis, Clin Radiol 63:59–70, 2008.
Jordan LC, Casella JF, DeBaun MR: Prospects for primary stroke prevention in children with sickle cell anaemia, Br J Haematol 157:14–25, 2012.
Kato GJ, Steinberg MH, Gladwin MT: Intravascular hemolysis and the pathophysiology of sickle cell disease, J Clin Invest 127:750–760, 2017.
King LGC, Bortolusso-Ali S, Cunningham CA, Reid MEG: Impact of a comprehensive sickle cell center on early childhood mortality in a developing country: the jamaican experience, J Pediatr 167:702–705, 2015.
Knight-Madden JM, Hambleton JR: Inhaled bronchodilators for acute chest syndrome in people with sickle cell disease, Cochrane Database Syst Rev (7):CD003733, 2012.
Kuhn JP, Slovis TL, Haller JO: Caffey’s pediatric diagnostic imaging, vol 2, ed 11, St Louis, 2008, Mosby, p 1087.
Kwiatkowski JL, Cohen AR, Garro J, et al: Transfusional iron overload in children with sickle cell anemia on chronic transfusion therapy for secondary stroke prevention, Am J Hematol 87:221–223, 2012.
Lehmann GC, Bell TR, Kirkham FJ, et al: Enuresis associated with sleep disordered breathing in children with sickle cell anemia, J Urol 188(4 Suppl):1572–1576, 2012.
Lettre G, Bauer DE: Feral haemoglobin in sickle-cell disease: from genetic epidemiology to new therapeutic strategies, Lancet 387:2554–2562, 2016.
Madigan C, Malik P: Pathophysiology and therapy for haemoglobinopathies. Part I. Sickle cell disease, Expert Rev Mol Med 8:1–23, 2006.
Mahadeo KM, Oyeku S, Taragin B, et al: Increased prevalence of osteonecrosis of the femoral head in children and adolescents with sickle-cell disease, Am J Hematol 86(9):806–808, 2011.
Mehari A, Gladwin MT, Tian X, et al: Mortality in adults with sickle cell disease and pulmonary hypertension, JAMA 307:1254–1256, 2012.
Meier ER, Miller JL: Sickle cell disease in children, Drugs 72:895–906, 2012.
Merritt AL, Haiman C, Henderson SO: Myth: blood transfusion is effective for sickle cell anemia-associated priapism, CJEM 8:119–122, 2006.
Miller AC, Gladwin MT: Pulmonary complications of sickle cell disease, Am J Respir Crit Care Med 185:1154–1165, 2012.
Miller ST, Kim HY, Weiner D, et al: Inpatient management of sickle cell pain: a «snapshot» of current practice, Am J Hematol 87:333–336, 2012.
Naik RP, Derebail VK, Grams ME, et al: Association of sickle cell trait with chronic kidney disease and albuminuria in African Americans, JAMA 312:2115–2124, 2014.
National Heart, Lung and Blood Institute: 2014 Expert Panel report on the evidencebased management of sickle cell disease. www.nhlbi.nih.gov/sites/www.nhlbi.nih.gov/files/sickle-cell-disease-report.pdf.
Nicholson GT, Hsu DT, Colan SD, et al: Coronary artery dilation in sickle cell disease, J Pediatr 159:789–794, 2011.
Niihara Y, Miller ST, Kanter J, et al: A phase 3 trial of L-glutamine in sickle cell disease, N Engl J Med 379(3):226–235, 2018.
Nordness ME, Lynn J, Zacharisen MC, et al: Asthma is a risk factor for acute chest syndrome and cerebral vascular accidents in children with sickle cell disease, Clin Mol Allergy 3:2, 2005.
Norris CF, Smith-Whitley K, McGowan KL: Positive blood cultures in sickle cell disease: time to positivity and clinical outcome, J Pediatr Hematol Oncol 25(5):390–395, 2003. Ohene-Frempong K, Weiner SJ, Sleeper LA, et al: Cerebrovascular accidents in sickle cell disease: rates and risk factors, Blood 91:288–294, 1998.
Panepinto JA, Bonner M: Health-related quality of life in sickle cell disease: past, present, and future, Pediatr Blood Cancer 59:377–385, 2012.
Panepinto JA, Brousseau DC, Hillery CA, et al: Variation in hospitalizations and hospital length of stay in children with vaso-occlusive crises in sickle cell disease, Pediatr Blood Cancer 44:182–186, 2005.
Parent F, Bachir D, Inamo J, et al: A hemodynamic study of pulmonary hypertension in sickle cell disease, N Engl J Med 365:44–52, 2011.
Piel FB, Patil AP, Howes RE, et al: Global epidemiology of sickle haemoglobin in neonates: a contemporary geostatistical model-based map and population estimates, Lancet 381:142–151, 2013.
Piel FB, Steinberg MH, Rees DC: Sickle cell disease, N Engl J Med 376(16):1561–1572, 2017.
Platt OS, Thorington BD, Brambilla DJ, et al: Pain in sickle cell disease: rates and risk factors, N Engl J Med 325(1):11–16, 1991.
Quillen K, Lane C, Hu E, et al: Prevalence of ceftriaxone-induced red blood cell antibodies in pediatric patients with sickle cell disease and human immunodeficiency virus infection, Pediatr Infect Dis J 27:357–358, 2008.
Quinn CT, McKinstry RC, Dowling MM, et al: Acute silent cerebral ischemic events in children with sickle cell anemia, JAMA Neurol 70:58–65, 2013.
Quinn CT, Rogers ZR, McCavit TL, et al: Improved survival of children and adolescents with sickle cell disease, Blood 115:3447–3452, 2010.
Reagan MM, DeBaun MR, Frei-Jones MJ: Multi-modal intervention for the inpatient management of sickle cell pain significantly decreases the rate of acute chest syndrome, Pediatr Blood Cancer 56:262–266, 2011.
Rezende PV, Viana MB, Murao M, et al: Acute splenic sequestration in a cohort of children with sickle cell anemia, J Pediatr (Rio J) 85:163–169, 2009.
Ribeil JA, Hacein-Bay-Abina S, Payen E, et al: Gene therapy in a patient with sickle cell disease, N Engl J Med 376(9):848–855, 2017.
Ridha A, Khan A, Al-Abayechi S, et al: Acute compartment syndrome secondary to rhabdomyolysis in a sickle cell trait patient, Lancet 384:2172, 2014.
Rogers ZR: Priapism in sickle cell disease, Hematol Oncol Clin North Am 19:917–928, viii, 2005.
Scothorn DJ, Price C, Schwartz D, et al: Risk of recurrent stroke in children with sickle cell disease receiving blood transfusion therapy for at least five years after initial stroke, J Pediatr 140:348–354, 2002.
Smith WR, Bauserman RL, Ballas SK, et al: Climatic and geographic temporal patterns of pain in the multicenter study of hydroxyurea, Pain 146:91–98, 2009.
Smith WR, Penberthy LT, Bovbjerg VE, et al: Daily assessment of pain in adults with sickle cell disease, Ann Intern Med 148:94–101, 2008.
Smith-Whitley K, Thompson AA: Indications and complications of transfusions in sickle cell disease, Pediatr Blood Cancer 59:358–364, 2012.
Smith-Whitley K, Zhao H, Hodinka RL, et al: Epidemiology of human parvovirus B19 in children with sickle cell disease, Blood 103:422–427, 2004.
Switzer JA, Hess DC, Nichols FT, et al: Pathophysiology and treatment of stroke in sickle-cell disease: present and future, Lancet Neurol 5:501–512, 2006.
Thornburg CD, Files BA, Luo Z, et al: Impact of hydroxyurea on clinical events in the BABY HUG trial, Blood 120:4304–4310, 2012.
Umans H, Haramati N, Flusser G: The diagnostic role of gadolinium enhanced MRI in distinguishing between acute medullary bone infarct and osteomyelitis, Magn Reson Imaging 18:255–262, 2000.
Vichinsky EP, Styles LA, Colangelo LH, et al: Acute chest syndrome in sickle cell disease: clinical presentation and course. Cooperative study of sickle cell disease, Blood 89(5):1787–1792, 1997.
Wang WC, Ware RE, Miller ST, et al: Hydroxycarbamide in very young children with sickle-cell anaemia: a multicentre, randomised, controlled trial (BABY HUG), Lancet 377:1663–1672, 2011.
Ware RE, Davis BR, Schultz WH, et al: Hydroxycarbamide versus chronic transfusion for maintenance of transcranial doppler flow velocities in children with sickle cell anaemia: TCD with transfusions changing to hydroxyurea (TWiTCH): a multicenter, open-label, phase 3, non-inferiority trial, Lancet 387:661–670, 2016.
Ware RE, de Montalembert M, Tshilolo L, Abboud MR: Sickle cell disease, Lancet 390:311–323, 2017.
Ware RE, Helms RW: Stroke with transfusions changing to hydroxyurea (SWiTCH), Blood 119:3925–3932, 2012.
Wilimas JA, Flynn PM, Harris S, et al: A randomized study of outpatient treatment with ceftriaxone for selected febrile children with sickle cell disease, N Engl J Med 329:472–476, 1993.
Yang YM, Shah AK, Watson M, et al: Comparison of costs to the health sector of comprehensive and episodic health care for sickle cell disease patients, Public Health Rep 110:80–86, 1995.
Nelson DA, Deuster PA, Carter IIIR, et al: Sickle cell trait, rhabdomyolysis, and mortality among US army soldiers, N Engl J Med 375(5):425–442, 2016.
Ambati SR, Randolph RE, Mennitt K, et al: Longitudinal monitoring of cardiac siderosis using cardiovascular magnetic resonance (CMR) T2* in patients with thalassemia major on various chelation regimens: a 6 year study, Am J Hematol 88:652–656, 2013.
Ash-Bernal R, Wise R, Wright SM: Acquired methemoglobinemia: a retrospective series of 138 cases at 2 teaching hospitals, Medicine (Baltimore) 83:265–273, 2004.
Baksi AJ, Pennell DJ: Randomized controlled trials of iron chelators for the treatment of cardiac siderosis in thalassemia major, Front Pharmacol 5:217, 2014.
Camaschella C: Treating iron overload, N Engl J Med 368:2325–2327, 2013.
Cash C, Arnold DH: Extreme methemoglobinemia after topical benzocaine: recognition by pulse oximetry, J Pediatr 181:319, 2017.
Chui DH, Fucharoen S, Chan V: Hemoglobin H disease: not necessarily a benign disorder, Blood 101:791–800, 2003.
Crowley MA, Mollan TL, Abdulmalik OY, et al: A hemoglobin variant associated with neonatal cyanosis and anemia, N Engl J Med 364:1837–1842, 2011.
Graff DM, Bosse GM, Sullivan J: Case report of methemoglobinemia in a toddler secondary to topical dapsone exposure, Pediatrics 138(2):e1–e2, 2016.
Higgs DR, Engel JD, Stamatoyannopoulos G: Thalassemia, Lancet 379:373–382, 2012.
Hussein AA, Al-Zaben A, Ghatasheh L, et al: Risk adopted allogeneic hematopoietic stem cell transplantation using a reduced intensity regimen for children with thalassemia major, Pediatr Blood Cancer 60:1345–1349, 2013.
Key NS, Derebail VK: Sickle-cell trait: novel clinical significance, Hematology Am Soc Hematol Educ Program 2010:418–422, 2010.
Kwiatkowski JL: Current recommendations for chelation for transfusion-dependent thalassemia, Ann N Y Acad Sci 1368:107–114, 2016.
Lal A, Goldrich ML, Haines DA, et al: Heterogeneity of hemoglobin H disease in childhood, N Engl J Med 364:710–718, 2011.
Martin A, Thompson AA: Thalassemias, Pediatr Clin North Am 60:1383–1391, 2013.
Musallam KM, Taher AT, Cappellini MD, et al: Clinical experience with fetal hemoglobin induction therapy in patients with beta-thalassemia, Blood 121:2199–2212, quiz 372, 2013.
Patel K, Livni N, Macdonald D: Renal medullary carcinoma, a rare cause of haematuria in sickle cell trait, Br J Haematol 132:1, 2006.
Peters M, Heijboer H, Smiers F, et al: Diagnosis and management of thalassaemia, BMJ 344:40–44, 2012.
Songdej D, Babbs C, Higgs DR: BHFS international consortium: an international registry of survivors with hb Bart’s hydrops fetalis syndrome, Blood 9:1251–1259, 2017.
Taher AT, Musallam KM, Karimi M, et al: Overview on practices in thalassemia intermedia management aiming for lowering complication rates across a region of endemicity: the OPTIMAL CARE study, Blood 115(10):1886–1892, 2010.
Taher AT, Musallam KM, Karimi M, et al: Overview on practices in thalassemia intermedia management aiming for lowering complication rates across a region of endemicity: the OPTIMAL CARE study, Blood 115:1886–1892, 2010.
Taher AT, Vichinsky E, Musallam KM, et al: Guidelines for the management of non transfusion dependent thalassaemia (NTDT), Nicosia, Cyprus, 2013, Thalassaemia Internation Federation.
Tsaras G, Owusu-Ansah A, Boateng FO, et al: Complications associated with sickle cell trait: a brief narrative review, Am J Med 122:507–512, 2009.
Vichinsky EP: Clinical manifestations of alpha-thalassemia, Cold Spring Harb Perspect Med 3:a011742, 2013.
Vichinsky E, Levine L: Standard of care guidelines for thalassemia, Oakland, CA, 2012, Children’s Hospital.
Vichinsky EP, MacKlin EA, Waye JS, et al: Changes in the epidemiology of thalassemia in north America: a new minority disease, Pediatrics 116:e818–e825, 2005.
Vichinsky E, Cohen A, Thompson AA, et al: Epidemiologic and clinical characteristics of nontransfusion-dependent thalassemia in the United States, Pediatr Blood Cancer 65(7):e27067, 2018.
Wood JC: Impact of iron assessment by MRI, Hematology Am Soc Hematol Educ Program 2011:443–450, 2011.
Aissa K, Kamoun F, Sfaihi L, et al: Hemolytic anemia and progressive neurologic impairment: think about triosephosphate isomerase deficiency, Fetal Pediatr Pathol 33:234–238, 2014.
Ayi K, Min-Oo G, Serghides L, et al: Pyruvate kinase deficiency and malaria, N Engl J Med 358:1805–1810, 2008.
Beutler E: Glucose-6-phosphate dehydrogenase deficiency, N Engl J Med 331:169–173, 1994.
Canu G, De Bonis M, Minucci A, Capoluongo E: Red blood cell PK deficiency: an update of PK-LR gene mutation database, Blood Cells Mol Dis 57:100–109, 2016.
Chiarelli LR, Morera SM, Bianchi P, et al: Molecular insights on pathogenic effects of mutations causing phosphoglycerate kinase deficiency, PLoS ONE 7:e32065, 2012.
Christensen RD, Nussenzveig RH, Yaish HM, et al: Causes of hemolysis in neonates with extreme hyperbilirubinemia, J Perinatol 34:616–619, 2014.
Climent F, Roset F, Repiso A, et al: Red cell glycolytic enzyme disorders caused by mutations: an update, Cardiovasc Hematol Disord Drug Targets 9:95–106, 2009. Coppens S, Koralkova P, Aeby A, et al: Recurrent episodes of myoglobinuria, mental retardation and seizures but no hemolysis in two brothers with phosphoglycerate kinase deficiency, Neuromuscul Disord 26:207–210, 2016.
Fujii H, Miwa S: Other erythrocyte enzyme deficiencies associated with nonhaematological symptoms: phosphoglycerate kinase and phosphofructokinase deficiency, Baillieres Best Pract Res Clin Haematol 13:141–148, 2000.
Gallagher PG: Diagnosis and management of rare congenital nonimmune hemolytic disease, Hematology Am Soc Hematol Educ Program 2015:392–399, 2015.
Gallagher PG, Glader B: Diagnosis of pyruvate kinase deficiency, Pediatr Blood Cancer 63:771–772, 2016. Garcia-Gomez M, Calabria A, Garcia-Bravo M, et al: Safe and efficient gene therapy for pyruvate kinase deficiency, Mol Ther 24:1187–1198, 2016.
Grace RF, Zanella A, Neufeld EJ, et al: Erythrocyte pyruvate kinase deficiency: 2015 status report, Am J Hematol 90:825–830, 2015.
Gunawardena S, Kapilananda GM, Samarakoon D, et al: Prevalence of G6PD deficiency in selected populations from two previously high malaria endemic areas of Sri Lanka, PLoS ONE 12(2):e0171208, 2017.
Howes RE, Battle KE, Satyagraha AW, et al: G6PD deficiency: global distribution, genetic variants and primaquine therapy, Adv Parasitol 81:133–201, 2013.
Kaplan M, Hammerman C, Bhutani VK: Parental education and the WHO neonatal G-6-PD screening program: a quarter century later, J Perinatol 35:779–784, 2015.
Lee SW, Lai NM, Chaiyakunapruk N, Chong DW: Adverse effects of herbal or dietary supplements in G6PD deficiency: a systematic review, Br J Clin Pharmacol 83:172–179, 2017.
Martinov MV, Plotnikov AG, Vitvitsky VM, et al: Deficiencies of glycolytic enzymes as a possible cause of hemolytic anemia, Biochim Biophys Acta 1474:75–87, 2000.
McMullin MF: The molecular basis of disorders of red cell enzymes, J Clin Pathol 52:241–244, 1999. Riskin A, Gery N, Kugelman A: Glucose-6-phosphate dehydrogenase deficiency and borderline deficiency: association with neonatal hyperbilirubinemia, J Pediatr 161:191–196, 2012.
Valentin C, Pissard S, Martin J, et al: Triose phosphate isomerase deficiency in 3 French families: two novel null alleles, a frameshift mutation (TPI Alfortville) and an alteration in the initiation codon (TPI Paris), Blood 96:1130–1135, 2000.
Van den Broek L, Heylen E, van den Akker M: Glucose-6-phosphate dehydrogenase deficiency: not exclusively in males, Clin Case Rep 4:1135–1137, 2016.
Veneri D, Facchinelli D, Vianello A, et al: Blood smear, a key diagnostic tool in hematology: lessons from two cases of acute hemolysis in previously undiagnosed G6PD deficiency, Am J Hematol 91:1165–1166, 2016.
Wong RJ, Stevenson DK: Neonatal hemolysis and risk of bilirubin-induced neurologic dysfunction, Semin Fetal Neonatal Med 20:26–30, 2015.
Zanella A, Fermo E, Bianchi P, et al: Red cell pyruvate kinase deficiency: molecular and clinical aspects, Br J Haematol 130:11–25, 2005.
Aladjidi N, Fernandes H, Leblanc T, et al: Evans syndrome in children: long-term outcome in a prospective French national observational cohort, Front Pediatr 3:79, 2015.
Aladjidi N, Leverger G, LeBlanc T, et al: New insights into childhood autoimmune hemolytic anemia: a French national observational study of 265 children, Haematologica 96:655–663, 2011.
Berentsen S, Ulvestad E, Gjertsen BT, et al: Rituximab for primary cold agglutinin disease: a prospective study of 37 courses of therapy in 27 patients, Blood 103: 2925–2928, 2004.
Dierickx D, Kentos A, Delannoy A: The role of rituximab in adults with warm antibody autoimmune hemolytic anemia, Blood 125:3223–3229, 2015.
Gehrs BC, Friedberg RC: Autoimmune hemolytic anemia, Am J Hematol 69:258–271, 2002. Gormezano NW, Kern D, Pereira OL, et al: Autoimmune hemolytic anemia in systemic lupus erythematosus at diagnosis: differences between pediatric and adult patients, Lupus 26:426–430, 2017.
Lechner K, J?ger U: How I treat autoimmune hemolytic anemias in adults, Blood 116:1831–1838, 2010.
Miano M: How I manage evans syndrome and AIHA cases in children, Br J Haematol 172:524–534, 2016.
Michel M: Classification and therapeutic approaches in autoimmune hemolytic anemia: an update, Expert Rev Hematol 4:607–618, 2011.
Price V: Auto-immune lymphoproliferative disorder and other secondary immune thrombocytopenias in childhood, Pediatr Blood Cancer 60(Suppl 1):S12–S14, 2013.
Sankaran J, Rodriguez V, Jacob EK, et al: Autoimmune hemolytic anemia in children: mayo clinic experience, J Pediatr Hematol Oncol 38:e120–e124, 2016.
Sove P, Bourdillon L, Sarrot-Reynauld F, et al: Autoimmune hemolytic anemia and common variable immunodeficiency, Medicine (Baltimore) 87:177–184, 2008.
Shanbhag S, Spivak J: Paroxysmal cold hemoglobinuria, Hematol Oncol Clin North Am 29:473–478, 2015.
Sparling TG, Andricevic M, Wass H: Remission of cold hemagglutinin disease induced by rituximab therapy, CMAJ 164:1405, 2001.
Teachey DT, Lambert MP: Diagnosis and management of autoimmune cytopenias in childhood, Pediatr Clin North Am 60:1489–1511, 2013.
Vagace JM, Bajo R, Gervasini G: Diagnostic and therapeutic challenges of primary autoimmune haemolytic anaemia in children, Arch Dis Child 99:668–673, 2014.
Woolley AE, Montgomery MW, Savage WJ, et al: Post-babesiosis warm autoimmune hemolytic anemia, N Engl J Med 376:939–946, 2017.
Zecca M, Nobili B, Ramenghi U, et al: Rituximab for the treatment of refractory autoimmune hemolytic anemia in children, Blood 101:3857–3861, 2003.
Bose S, Sonny A, Rahman N: A teenager presents with fulminant hepatic failure and acute hemolytic anemia, Chest 147:e100–e104, 2015.
George JN, Nester CM: Syndromes of thrombotic microangiopathy, N Engl J Med 371:654–666, 2014.
Hedera P: Update on the clinical management of Wilson’s disease, Appl Clin Genet 10:9–19, 2017.
Riedl M, Fakhouri F, Le Quintrec M, et al: Spectrum of complement-mediated thrombotic microangiopathies: pathogenetic insights identifying novel treatment approaches, Semin Thromb Hemost 40:444–464, 2014.
Trachtman H: HUS and TTP in children, Pediatr Clin North Am 60:1513–1526, 2013.
Ye XN, Mao LP, Lou YJ, Tong HY: Hemolytic anemia as first presentation of Wilson’s disease
Barbui T: How to manage children and young adults with myeloproliferative neoplasms, Leukemia 26(7):1452–1457, 2012.
Cario H, McMullin MF, Bento C, et al: Erythrocytosis in children and adolescents: classification, characterization, and consensus recommendations for the diagnostic approach, Pediatr Blood Cancer 60:1734–1738, 2013.
Cario H, McMullin MF, Pahl HL: Clinical and hematological presentation of children and adolescents with polycythemia vera, Ann Hematol 88(8):713–719, 2009.
Cario H, Schwarz K, Herter JM, et al: Clinical and molecular characterization of a prospectively collected cohort of children and adolescents with polycythemia vera, Br J Haematol 142:622–626, 2008.
Cazzola M: Introduction to a review series on myeloproliferative neoplasms, Blood 129:667–703, 2017.
Geyer H, Scherber R, Kosiorek H, et al: Symptomatic profiles of patients with polycythemia vera: implications of inadequately controlled disease, J Clin Oncol 34(2):151–159, 2016.
Hofmann I: Myeloproliferative neoplasms in children, J Hematop 8(3):143–157, 2015.
Karow A, Nienhold R, Lundberg P, et al: Mutational profile of childhood myeloproliferative neoplasms, Leukemia 29:2407–2409, 2015.
Patnaik MM, Tefferi A: The complete evaluation of erythrocytosis: congenital and acquired, Leukemia 23:834–844, 2009.
Pemmaraju N, Mesa R: Evidence for janus kinase (JAK) inhibitors for the prevention of major morbid events in patients with myeloproliferative neoplasms (MPNs), Hematology Am Soc Hematol Educ Program 649–651:2015, 2015.
Radia D, Geyer HL: Management of symptoms in polycythemia vera and essential thrombocythemia patients, Hematology Am Soc Hematol Educ Program 2015:340–348, 2015.
Spivak JL, Silver RT: The revised world health organization diagnostic criteria for polycythemia vera, essential thrombocytosis, and primary myelofibrosis: an alternative proposal, Blood 112(2):231–239, 2008.
Tefferi A: Myeloproliferative neoplasms: a decade of discoveries and treatment advances, Am J Hematol 91(1):50–58, 2016.
Teofili L, Foa R, Giona F, et al: Childhood polycythemia vera and essential thrombocytopenia: does their pathogenesis overlap with that of adult patients?, Haematologica 93:169–172, 2008.
Teofili L, Giona F, Martini M, et al: Markers of myeloproliferative diseases in childhood polycythemia vera and essential thrombocythemia, J Clin Oncol 25:1048–1053, 2007.
Alsafadi TR, Hashmi SM, Youssef HA, et al: Polycythemia in neonatal intensive care unit: risk factors, symptoms, pattern, and management controversy, J Clin Neonatol 3(2):93–98, 2014.
Cario H, McMullin MF, Bento C, et al: Erythrocytosis in children and adolescents: classification, characterization, and consensus recommendations for the diagnostic approach, Pediatr Blood Cancer 60(11):1734–1738, 2013.
Chauveau A, Luque Paz D, Lecucq L, et al: A new point mutation in EPOR inducing a short deletion in congenital erythrocytosis, Br J Haematol 172(3):475–477, 2016.
Giona F, Teofili L, Moleti ML, et al: Thrombocythemia and polycythemia in patients younger than 20 years at diagnosis: clinical and biologic features, treatment, and long-term outcome, Blood 119(10):2219–2227, 2012.
Huang LJ, Shen YM, Bulut GB: Advances in understanding the pathogenesis of primary familial and congenital polycythaemia, Br J Haematol 148(6):844–852, 2010.
Pagon RA, Adam MP, Ardinger HH, et al: Primary familial and congenital polycythemia. In GeneReviews [Internet], Seattle, 1993–2017, University of Washington.
Remon JI, Raghavan A, Maheshwari A: Polycythemia in the newborn, Neoreviews 12:e20, 2011.
Sergueeva AI, Miasnikova GY, Polyakova LA, et al: Complications in children and adolescents with chuvash polycythemia, Blood 125(2):414–415, 2015.
Sidhu A, Bhambhani K, Callaghan MU: Novel mutations in the von Hippel–lindau gene associated with congenital polycythemia, Pediatr Blood Cancer 62(6):1113–1114, 2015.
Siehr SL, Shi S, Hao S, et al: Exploring the role of polycythemia in patients with cyanosis after palliative congenital heart surgery, Pediatr Crit Care Med 17(3):216–222, 2016.
Watchko JF: Common hematologic problems in the newborn nursery, Pediatr Clin North Am 62(2):509–524, 2015.
Alter BP, Giri N, Savage SA, et al: Malignancies and survival patterns in the national cancer institute inherited bone marrow failure syndromes cohort study, Br J Haematol 150:179–188, 2010.
Ballmaier M, Germeshausen M: Congenital amegakaryocytic thrombocytopenia: clinical presentation, diagnosis, and treatment, Semin Thromb Hemost 37:673–681, 2011.
Cada M, Segbefia C, Klaassen R, et al: The impact of category, cytopathology and cytogenetics on development and progression of clonal and malignant myeloid transformation in inherited bone marrow failure syndromes, Haematologica 100:633–642, 2015.
Dror Y, Donadieu J, Koglmeier J, et al: Draft consensus guidelines for diagnosis and treatment of Shwachman-diamond syndrome, Ann NY Acad Sci 1242:40–55, 2011.
Dufour C: How I manage patients with fanconi anemia, Br J Haematol 178:32–47, 2017.
Ghemlas I, Li H, Zlateska B, et al: Improving diagnostic precision, care and syndrome definitions using comprehensive next-generation sequencing for the inherited bone marrow failure syndromes, J Med Genet 52:575–584, 2015.
Gueiderikh A, Rosselli F, Neto JBC: A never-ending story: the steadily growing family of the FA and FA-like genes, Genet Mol Biol 40:398–407, 2017.
Kottemann MC, Smogorzewska A: Fanconi anaemia and the repair of watson and crick DNA crosslinks, Nature 493:356–363, 2013.
Paustian L, Chao MM, Hanenberg H, et al: Androgen therapy in fanconi anemia: a retrospective analysis of 30 years in Germany, Pediatr Hematol Oncol 33:5–12, 2016.
Savage SA: Dyskeratosis congenita, synonym: Zinsser-cole-engman syndrome, GeneReviews 2016.
Shimamura A, Alter BP: Pathophysiology and management of inherited bone marrow failure syndromes, Blood Rev 24:101–122, 2010.
Tsangaris E, Adams SL, Yoon G, et al: Ataxia and pancytopenia caused by a mutation in TINF2, Hum Genet 124:507–513, 2008.
Wegman-Ostrosky T, Savage SA: The genomics of inherited bone marrow failure: from mechanism to the clinic, Br J Haematol 177:526–542, 2017.
Weis F, Giudice E, Churcher M, et al: Mechanism of eIF6 release from the nascent 60s ribosomal subunit, Nat Struct Mol Biol 22:914–919, 2015.
Babushok D, Bessler M, Olson T: Genetic predisposition to myelodysplastic syndrome and acute myeloid leukemia in children and young adults, Leuk Lymphoma 57:520–536, 2016.
Barone A, Lucarelli A, Onofrillo D, et al: Diagnosis and management of acquired aplastic anemia in childhood: guidelines from the marrow failure study group of the pediatric Haemato-oncology Italian association (AIEOP), Blood Cells Mol Dis 55:40–47, 2015.
Chung NG, Lee JW, Jang PS, et al: Reduced dose cyclophosphamide, fludarabine, and antithymocyte globulin for sibling and unrelated transplant of children with severe and very severe aplastic anemia, Pediatr Transplant 17:387–393, 2013.
Desmond R, Townsley DM, Dumitriu B, et al: Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug, Blood 123:1818–1825, 2014.
Im HJ, Koh KN, Choi ES, et al: Excellent outcome of haploidentical hematopoietic stem cell transplantation in children and adolescents with acquired severe aplastic anemia, Biol Blood Marrow Transplant 19:754–759, 2013.
Ngwube A, Hayashi RJ, Murray L, et al: Alemtuzumab based reduced intensity transplantation for pediatric severe aplastic anemia, Pediatr Blood Cancer 62:1270–1276, 2015.
Scheinberg P: Prognostic value of telomere attrition in patients with aplastic anemia, Int J Hematol 97:553–557, 2013.
Shallis RM, Ahmad R, Zeidan AM: Aplastic anemia: etiology, molecular pathogenesis, and emerging concepts, Eur J Haematol 101:711–720, 2018.
Townsley DM, Scheinberg P, Winkler T, et al: Eltrombopag added to standard immunosuppression for aplastic anemia, N Engl J Med 376(16):1540–1550, 2017.
Tsoukas MA: Pancytopenia in severe hypothyroidism, Am J Med 127(9):e11–e12, 2014.
Yoshimi A, Niemeyer CM, Fuhrer MM, et al: Comparison of the efficacy of rabbit and horse antithymocyte globulin for the treatment of severe aplastic anemia in children, Blood 121:860–861, 2013.
Yoshizato T, Dumitriu B, Hosokawa K, et al: Somatic mutations and clonal hematopoiesis in aplastic anemia, N Engl J Med 373:35–46, 2015.
Bell EF, Strauss RG, Widness JA, et al: Randomized trial of liberal versus restrictive guidelines for red blood cell transfusion in preterm infants, Pediatrics 115:1685–1691, 2005.
Bergman FS, Almeida JP, Landoni G, et al: Liberal versus restrictive transfusion strategy in critically ill oncologic patients: the transfusion requirements in critically ill oncologic patients randomized controlled trial, Crit Care Med 45(5):766–773, 2017.
Carson JL, Carless PA, H?bert PC: Outcomes using lower vs. higher hemoglobin thresholds for red blood cell transfusion, JAMA 309(1):83–84, 2013.
Fergusson DA, H?bert P, Hogan DL, et al: Effect of fresh red blood transfusions on clinical outcomes in premature, very low-birth-weight infants: the ARIPI randomized trial, JAMA 308:1443–1451, 2012.
Goel R, Cushing MM, Tobian AR: Pediatric patient blood management programs: not just transfusing little adults, Trans Med Rev 30:235–241, 2016.
Goodnough LT, Levy JH, Murphy MF: Concepts of blood transfusions in adults, Lancet 381:1845–1852, 2013.
Hobert PC, McDonald BJ, Tinmouth A: Clinical consequences of anemia and red cell transfusion in the critically ill, Crit Care Clin 20:225–235, 2004.
Hendrickson JE, Shaz BH, Pereira G, et al: Implementation of a pediatric trauma massive transfusion protocol: a quality improvement initiative, Transfusion 52:1228–1236, 2012.
Hwu RS, Spinella PC, Keller MS, et al: The effect of massive transfusion protocol implementation on pediatric trauma care, Transfusion 56:2712–2719, 2016.
Josephson CD, Caliendo AM, Easley KA, et al: Blood transfusion and breast milk transmission of cytomegalovirus in very-low-birth-weight infants: a prospective cohort study, JAMA Pediatr 168:1054–1062, 2014.
Kirpalani H, Whyte RK, Andersen C, et al: The premature infants in need of transfusion (PINT) study: a randomized, controlled trial of a restrictive (low) versus liberal (high) transfusion threshold for extremely low birth weight infants, J Pediatr 149:301–307, 2006.
Lacroix J, H?bert PC, Hutchison JS, et al: Transfusion strategies for patients in pediatric intensive care units, N Engl J Med 356:1609–1619, 2007.
Strauss RG: Controversies in the management of the anemia of prematurity using single-donor red blood cell transfusion and/or recombinant human erythropoietin, Transfuse Med Rev 20:34–44, 2006.
Strauss RG: Commentary: is it safe to limit allogeneic red blood cell transfusions to neonates? Neonatology 93:217–222, 2008.
Strauss RG: How I transfuse red blood cells and platelets to infants, Transfusion 48:209–217, 2008.
Strauss RG: Anaemia of prematurity: pathophysiology and treatment, Blood Rev 24:221–225, 2010.
Vamvakas EC, Strauss RG: Meta-analysis of controlled clinical trials studying the efficacy of recombinant human erythropoietin in reducing blood transfusions in the anemia of prematurity, Transfusion 41:406, 2001.
Venkatesh V, Khan R, Curley A, et al: The safety and efficacy of red cell transfusions in neonates: a systematic review of randomized controlled trials, Br J Haematol 158:370–385, 2012.
Andrew M, Vegh P, Caco C, et al: A randomized controlled trial of platelet transfusions in thrombocytopenic premature infants, J Pediatr 123:285–291, 1993.
Baer VL, Lambert DK, Henry E, et al: Severe thrombocytopenia in the NICU, Pediatrics 124:1095–1100, 2009.
Christensen RD, Paul DA, Sola-Visner MC, et al: Improving platelet transfusion practices in the neonatal intensive care unit, Transfusion 48:2281–2284, 2008.
Curley A, Stanworth SJ, Willoughby K, et al: Randomized trial of platelet transfusion thresholds in neonates, N Engl J Med 2018. doi:10.1056/NEJMoa1807320.
Ferrer-Marin F, Liu ZJ, Gutti R, et al: Neonatal thrombocytopenia and megakaryocytopoiesis, Semin Hematol 47:281–288, 2010.
Josephson CD, Granger S, Assmann SF, et al: Bleeding risks are higher in children versus adults given prophylactic platelet transfusions for treatment-induced hypoproliferative thrombocytopenia, Blood 120:748–760, 2012.
Josephson CD, Su LL, Christensen RD, et al: Platelet transfusion practices among neonatologists in the United States and Canada: results of a survey, Pediatrics 123:278–285, 2009.
Josephson CD, Caliendo AM, Easley KA, et al: Blood transfusion and breast milk transmission of cytomegalovirus in very-low-birth-weight infants: a prospective cohort study, JAMA Pediatr 168:1054–1062, 2014.
Sparger KA, Assmann SF, Granger S, et al: Platelet transfusion practices among very-low-birth-weight infants, JAMA Pediatr 170:687–694, 2016.
Strauss RG: Platelet transfusion volume reduction: it can be done, but why do it?, Transfusion 53:3029–3031, 2013.
Strauss RG: Platelet transfusion in neonates and children. In Sweeney JD, Lozano M, editors: Platelet transfusion therapy, Bethesda, MD, 2013, AABB Press, pp 354–369.
Stanworth SJ, Grant-Casey J, Lowe D, et al: The use of fresh-frozen plasma in England: high levels of inappropriate use in adults and children, Transfusion 51:62–70, 2011.
Strauss RG: Blood components for infants. In Blajchman M, Cid J, Lonzano M, editors: Blood component preparation: from benchtop to bedside, Bethesda, MD, 2011, AABB Press, pp 211–246.
Keir AK, Stanworth SJ: Neonatal plasma transfusion: an evidenced-based review, Transfus Med Rev 30:174–182, 2016.
Stanworth SJ, Grant-Casey J, Lowe D, et al: The use of fresh-frozen plasma in England: high levels of inappropriate use in adults and children, Transfusion 51:62–70, 2011.
Strauss RG: Blood components for infants. In Blajchman M, Cid J, Lonzano M, editors: Blood component preparation: from benchtop to bedside, Bethesda, MD, 2011, AABB Press, pp 211–246.
Venkatesh V, Khan R, Curley A, et al: How we decide when a neonate needs a transfusion, Br J Haematol 160:421–433, 2013.
Alter HJ, Kein HG: The hazards of blood transfusion in historical perspective, Blood 112:2617–2626, 2008.
Azarow KS: Use of fresh frozen plasma in children, J Pediatr 160(2):185–186, 2012.
Blood: a precious resource, Lancet 381:2013, 1789.
Caram-Deelder C, Krueger AL, Evers D, et al: Association of blood transfusion from female donors with and without a history of pregnancy with mortality among males and female transfusion recipients, JAMA 318(15):1471–1478, 2017.
Carson JL, Triulzi DJ, Ness PM: Indications for and adverse effects of red-cell transfusion, N Engl J Med 377(13):1261–1272, 2017.
Centers for Disease Control and Prevention: Blood donor screening for chagas disease — United States, 2006–2007, MMWR 56:141–143, 2007.
Centers for Disease Control and Prevention: Anaplasma phagocytophilum transmitted through blood transfusion — minnesota, 2007, MMWR 57:1145–1148, 2008.
Centers for Disease Control and Prevention: West nile virus transmission via organ transplantation and blood transfusion — louisiana, 2008, MMWR 58:1263–1266, 2009.
Centers for Disease Control and Prevention: Transfusion-related transmission of yellow fever vaccine virus — california, 2009, MMWR 59:34–36, 2010.
Cooper DJ, McQuilten ZK, Nichol A, et al: Age of red cells for transfusion and outcomes in critically ill adults, N Engl J Med 377(19):1858–1867, 2017.
Delaney M, Wendel S, Bercovitz RS: Transfusion reactions: prevention, diagnosis, and treatment, Lancet 388:2825–2836, 2016.
Goodnough LT: Blood management: transfusion medicine comes of age, Lancet 381:1791–1792, 2013.
Hendrickson JE, Shaz BH, Pereira G, et al: Coagulopathy is prevalent and associated with adverse outcomes in transfused pediatric trauma patients, J Pediatr 160:204–209, 2012.
Hladik W, Dollard SC, Mermin J, et al: Transmission of human herpesvirus 8 by blood transfusion, N Engl J Med 355:1331–1338, 2006.
Jackups R, Savage W: Gaps in research on adverse events to transfusion in pediatrics, Transfus Med Rev 30:209–212, 2016.
Jimenez A, Shaz BH, Bloch EM: Zika virus and the blood supply: what do we know?, Transfus Med Rev 31:1–10, 2016.
Josephson CD, Caliendo AM, Easley KA, et al: Blood transfusion and breast milk transmission of cytomegalovirus in very-low-birth-weight infants: a prospective cohort study, JAMA Pediatr 168:1054–1062, 2014.
Kekre N, Tokessy M, Mallick R, et al: Is cytomegalovirus testing of blood products still needed for hematopoietic stem cell recipients in the era of universal leukoreduction?, Biol Blood Marrow Trans 19:1719–1724, 2013.
Rohde JM, Dimcheff DE, Blumberg N, et al: Health care–associated infection after red blood cell transfusion — a systematic review and meta-analysis, JAMA 311:1317–1326, 2014.
Simonsen KA, Harwell JI, Lainwala S: Clinical presentation and treatment of transfusion-associated babesiosis in premature infants, Pediatrics 128(4):e1019–e1024, 2011.
Zieman M, Heuft H-G, Frank K, et al: Window period donations during primary cytomegalovirus infection and risk of transmitted-transmitted infections, Transfusion 53:1088–1109, 2013.
Zieman M, Krueger S, Maier AB, et al: High prevalence of cytomegalovirus DNA in plasma samples of blood donors in connection with seroconversion, Transfusion 47:1972–1983, editorial, 1955–1958, 2007.
Boender J, Kruip MJ, Leebeek FW: A diagnostic approach to mild bleeding disorders, J Thromb Haemost 14:1507–1516, 2016.
Branchford B, Di Paola J: Approach to the child with a suspected bleeding disorder. In Orkin SH, Fisher DE, Ginsberg D, et al, editors: Nathan and Oski’s hematology of infancy and childhood, ed 8, Philadelphia, 2015, Saunders Elsevier, pp 951–963.
Cantor AB: Hemostasis in the newborn and infant. In Nathan and Oski’s hematology of infancy and childhood, ed 8, Philadelphia, 2015, Saunders Elsevier, pp 128–157.
Jaffray J, Young G, Ko RH: The bleeding newborn: a review of presentation, diagnosis, and management, Semin Fetal Neonatal Med 21:44–49, 2016.
Levi M, van der Poll T: Coagulation and sepsis, Thromb Res 149:38–44, 2017.
Harrison P: The role of PFA-100 testing in the investigation and management of haemostatic defects in children and adults, Br J Haematol 130:3–10, 2005.
Lippi G, Franchini M, Montagnana M, et al: Coagulation testing in pediatric patients: the young are not just miniature adults, Semin Thromb Hemost 33:816–820, 2007.
Mann KG, Brummel-Ziedens K: Blood coagulation. In Nathan and Oski’s hematology of infancy and childhood, ed 8, Philadelphia, 2015, Saunders Elsevier, pp 964–983.
Newman DK, Newman PJ: Platelets and the vessel wall. In Nathan and Oski’s hematology of infancy and childhood, ed 8, Philadelphia, 2015, Saunders Elsevier, pp 951–963.
Parmar N, Albisetti M, Berry LR, et al: The fibrinolytic system in newborns and children, Clin Lab 52:115–124, 2006. erratum, 52:324, 2006.
Saxonhouse MA, Manco-Johnson MJ: The evaluation and management of neonatal coagulation disorders, Semin Perinatol 33:52–65, 2009.
Tomaiulo M, Brass LF, Stalker TJ: Regulation of platelet activation and coagulation and its role in vascular injury and arterial thrombosis, Interv Cardiol Clin 6:1–12, 2017.
Versteeg HH, Heemskerk JWM, Levi M, et al: New fundamentals in hemostasis, Physiol Rev 93:327–358, 2013.
Aledort LM: The evolution of comprehensive haemophilia care in the United States: perspectives from the frontline, Haemophilia 22:676–683, 2016.
Berntorp E, Shapiro AD: Modern haemophilia care, Lancet 379:1447–1454, 2012.
Branchford BR, Monahan PE, Di Paola J: New developments in the treatment of pediatric hemophilia and bleeding disorders, Curr Opin Pediatr 25:23–30, 2013.
Broderick CR, Herbert RD, Latimer J, et al: Association between physical activity and risk of bleeding in children with hemophilia, JAMA 308:1452–1458, 2012.
Coppola A, Tagliaferri A, Di Capua M, et al: Prophylaxis in children with hemophilia: evidence-based achievements, old and new challenges, Semin Thromb Hemost 38(1):79–94, 2012.
Di Paola J, Montgomery RR, Gill JC, Flood VH: Hemophilia and von willebrand disease. In Orkin SH, Fisher DE, Ginsberg D, et al, editors: Nathan and Oski’s hematology of infancy and childhood, ed 8, Philadelphia, 2015, Saunders Elsevier, pp 1028–1054.
George LA, Fogarty PF: Gene therapy for hemophilia: past, present, and future, Semin Hematol 53:46–54, 2016.
Gouw SC, van der Bom JG, Ljung R, et al: Factor VIII products and inhibitor development in severe hemophilia a, N Engl J Med 368:231–238, 2013.
Kulkarni R, Soucie JM, Lusher J, et al: Site of initial bleeding episodes, mode of delivery and age of diagnosis in babies with haemophilia diagnosed before the age of 2 years: a report from the centers for disease control and Prevention’s (CDC) universal data collection (UDC) project, Haemophilia 15:1281–1290, 2009.
Kumar R, Dunn A, Carcao M: Changing paradigm of hemophilia management: extended half-life concentrates and gene therapy, Semin Thromb Hemost 42:18–29, 2016.
Leissinger CA: Advances in the clinical management of inhibitors in hemophilia a and B, Semin Hematol 53:20–27, 2016.
Manco-Johnson MJ, Abshire TC, Shapiro AD, et al: Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia, N Engl J Med 357:535–544, 2007.
Manco-Johnson MJ: Collision sports and risk of bleeding in children with hemophilia, JAMA 308:1480–1481, 2012.
Manco-Johnson MJ, Soucie JM, Gill JC: Prophylaxis usage, bleeding rates and joint outcomes of hemophilia 1999–2019: a surveillance project, Blood 129(17):2368–2374, 2017.
The Medical Letter: Eloctate for hemophilia a, Med Lett 57(1479):143–144, 2015.
Nathwani AC, Reiss UM, Tuddenham EGD, et al: Long-term safety and efficacy of factor IX gene therapy in hemophilia B, N Engl J Med 371(21):1994–2004, 2014.
Nathwani AC, Tuddenham EGD, Rangarajan S, et al: Adenovirus-associated virus vector-mediated gene transfer in hemophilia B, N Engl J Med 365:2357–2365, 2011.
Oldenburg J, Mahlangu JN, Kim B, et al: Emicizumab prophylaxis in hemophilia a with inhibitors, N Engl J Med 377(9):809–818, 2017.
Pasi KJ, Rangarajan S, Georgiev P, et al: Targeting of antithrombin in hemophilia a or B with RNAi therapy, N Engl J Med 377(9):819–828, 2017.
Peyvandi F, Ettingshausen CE, Goudemand J, et al: New findings on inhibitor development: from registries to clinical studies, Haemophilia 23(Suppl 1):4–13, 2017.
Peyvandi F, Garagiola I, Young G: The past and future of haemophilia: diagnosis, treatments, and its complications, Lancet 388:187–197, 2016.
Peyvandi F, Mannucci PM, Garagiola I, et al: A randomized trial of factor VIII and neutralizing antibodies in hemophilia a, N Engl J Med 374(21):2054–2064, 2016.
Pipe SW: New therapies for hemophilia, Hematology Am Soc Hematol Educ Program 2016:650–656, 2016.
Santagostino E, Martinowitz U, Lissitchkov T, et al: Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial, Blood 127(14):1761–1769, 2016.
Shima M, Hanabusa H, Taki M, et al: Factor VIII-mimetic function of humanized bispecific antibody in hemophilia a, N Engl J Med 374(21):2044–2052, 2016.
Thalji N, Camire RN: Parahemophilia: new insights into factor V deficiency, Semin Thromb Hemost 39:607–612, 2013.
Zheng C, Zhang B: Combined deficiency of coagulation factors V and VIII: an update, Semin Thromb Hemost 39:613–620, 2013.
Bornikova L, Peyvandi F, Allen G, et al: Fibrinogen replacement therapy for congenital fibrinogen deficiency, J Thromb Haemost 9:1687–1704, 2011.
De Moerloose P, Casini A, Neerman-Arbez M: Congenital fibrinogen disorders: an update, Semin Thromb Hemost 39:585–595, 2013.
Biswas A, Ivaskevicius V, Thomas A, Oldenburg J: Coagulation factor XIII deficiency: diagnosis, prevalence, and management of inherited and acquired forms, Haemostasiologie 34:160–166, 2014.
Dorgalaleh A, Rashidpanah J: Blood coagulation factor XIII and factor XIII deficiency, Blood Rev 30:461–475, 2016.
Dreyfus M, Barrois D, Borg JY, et al: Successful long-term replacement therapy with FXIII concentrate (fibrogammin p) for severe congenital factor XIII deficiency: a prospective multicentre study, J Thromb Haemost 9(6):1264–1266, 2011.
Bauer KA: Rare coagulation factor abnormalities. In Orkin SH, Nathan DG, Ginsberg D, et al, editors: Nathan and Oski’s hematology of infancy and childhood, ed 7, Philadelphia, 2009, Saunders Elsevier, pp 1525–1532.
Carpenter SL, Mathew P: Alpha2-antiplasmin and its deficiency: fibrinolysis out of balance, Haemophilia 14:1250–1254, 2008.
Di Paola J, Montgomery RR, Gill JC, et al: Hemophilia and von willebrand disease. In Orkin SH, Fisher DE, Ginsberg D, editors: Nathan and Oski’s hematology of infancy and childhood, ed 8, Philadelphia, 2015, Saunders Elsevier, pp 1028–1054.
Eikenboom J, Federici AB, Dirven RJ, et al: VWF propeptide and ratios between VWF, VWF propeptide, and FVIII in the characterization of type 1 von willebrand disease, Blood 121:2336–2339, 2013.
Federici AB: Current and emerging approaches for assessing von willebrand disease in 2016, Int J Lab Hematol 38(Suppl 1):41–49, 2016.
Goodeve AC: The genetic basis of von willebrand disease, Blood Rev 24:123–135, 2010. James AH, Eikenboom J, Federici AB: State of the art: von willebrand disease, Haemophilia 22(Suppl 5):54–59, 2016.
Laffan MA, Lester W, O’Donnell JS, et al: The diagnosis and management of von willebrand disease: a United Kingdom haemophilia centre doctors organization guideline approved by the British committee for standards in haematology, Br J Haematol 167:453–465, 2014.
Leebeek FW, Eikenboom JC: Von Willebrand’s disease, N Engl J Med 375:2067–2080, 2016.
Mannucci PM, Federici AB, James AH, et al: Von willebrand disease in the 21st century: current approaches and new challenges, Haemophilia 15:1154–1158, 2009.
Nichols WL, Hultin MB, James AH, et al: Von willebrand disease (VWD): evidencebased diagnosis and management guidelines, the national heart, Lung and blood institute (NHLBI) expert panel report (USA), Haemophilia 14:171–232, 2008.
Andrew M, Paes B, Milner R, et al: Development of the human coagulation system in the full-term infant, Blood 70:165–172, 1987.
Bertina RM, Koeleman BP, Koster T, et al: Mutation in blood coagulation factor V associated with resistance to activated protein C, Nature 369:64–67, 1994.
Bezemer ID, Bare LA, Doggen CJM, et al: Gene variants associated with deep vein thrombosis, JAMA 299:1306–1314, 2008.
Connors JM: Thrombophilia testing and venous thrombosis, N Engl J Med 377(12):1177–1186, 2017.
Coppola A, Tufano A, Cerbone AM, et al: Inherited thrombophilia: implications for prevention and treatment of venous thromboembolism, Semin Thromb Hemost 35:683–694, 2009.
Hicks LK, Bering H, Carson KR, et al: The ASH choosing wisely campaign: five hematologic tests and treatments to question, Blood 122:3879–3883, 2013.
Holzhauer S, Goldenberg NA, Junker R, et al: Inherited thrombophilia in children with venous thromboembolism and the familial risk of thromboembolism: an observational study, Blood 120:1510–1515, 2012.
Kreuz W, Stoll M, Junker R, et al: Familial elevated factor VIII in children with symptomatic venous thrombosis and post-thrombotic syndrome: results of a multicenter study, Arterioscler Thromb Vasc Biol 26:1901–1906, 2006.
MacCallum P: Diagnosis and management of heritable thrombophilias, BMJ 349:g4387, 2014.
Martinelli I, Bucciarelli P, Artoni A, et al: Anticoagulant treatment with rivaroxaban in severe protein S deficiency, Pediatrics 132(5):e1435–e1439, 2013.
Middeldorp S, van Hylckama Vlieg A: Does thrombophilia testing help in the clinical management of patients?, Br J Haematol 143:321–335, 2008.
Monagle P, Barnes C, Ignjatovic V, et al: Developmental haemostasis: impact for clinical haemostasis laboratories, Thromb Haemost 95:362–372, 2006.
Neshat-Vahid S, Pierce R, Hershey D, et al: Association of thrombophilia and catheter associated thrombosis in children: a systematic review and meta-analysis, J Thromb Haemost 14:1749–1758, 2016.
Raffini L: Thrombophilia in children: who to test, how, when, and why?, Hematology Am Soc Hematol Educ Program 2008:228–235, 2008.
Revel-Vilk SG: Thrombophilia in children with venous thromboembolic disease, Thromb Res 118:59–65, 2006.
Segal JB, Brotman DJ, Necochea AJ, et al: Predictive value of factor V Leiden and prothrombin G20210A in adults with venous thromboembolism and in family members of those with a mutation, JAMA 301:2472–2484, 2009.
Stevens SM, Woller SC, Bauer KA, et al: Guidance for the evaluation and treatment of hereditary and acquired thrombophilia, J Thromb Thrombolysis 41:154–164, 2016.
Thornburg CD, Dixon N, Paulyson-Nunez K, et al: Thrombophilia screening in asymptomatic children, Thromb Res 121:597–604, 2008.
Yang JYK, Chan AKC: Pediatric thrombophilia, Pediatr Clin North Am 60:1443–1462, 2013.
Young G, Albisetti M, Bonduel M, et al: Impact of inherited thrombophilia on venous thromboembolism in children, Circulation 118:1373–1382, 2008.
Alberts MJ: Genetic of warfarin dosing: one polymorphism at a time, Lancet 382:749–751, 2013.
Ansah DA, Patel KN, Montegna L, et al: Tissue plasminogen activator use in children: bleeding complications and thrombus resolution, J Pediatr 171:67–72, 2016.
Baskin JL, Pui CH, Reiss U, et al: Management of occlusion and thrombosis associated with long-term indwelling central venous catheters, Lancet 374:159–169, 2009.
Branduo LR, Williams S, Kahr WHA, et al: Exercise-induced deep vein thrombosis of the upper extremity, Acta Haematol 115:221–229, 2006.
Boller HR, ten Cate-Hoek AJ, Hoes AW, et al: Safely ruling out deep venous thrombosis in primary care, Ann Intern Med 150:229–235, 2009.
Castellucci LA, Cameron C, Le Gal G, et al: Clinical and safety outcomes associated with treatment of acute venous thromboembolism: a systematic review and metaanalysis, JAMA 312:1122–1134, 2014.
Chang K, Graf E, Davis K, et al: Spectrum of thoracic outlet syndrome presentation in adolescents, Arch Surg 146:1383–1387, 2011.
Chopra V, Anand S, Hickner A, et al: Risk of venous thromboembolism associated with peripherally inserted central catheters: a systematic review and meta-analysis, Lancet 382:311–324, 2013.
Cohen AT, Harrington RA, Goldhaber SZ, et al: Extended thromboprophylaxis with betrixaban in acutely ill medical patients, N Engl J Med 375(6):534–544, 2016.
Di Nisio M, van Es N, B?ller HR: Deep vein thrombosis and pulmonary embolism, Lancet 388:3060–3069, 2016.
Eikelboom JW, Weitz JI: Selective factor xa inhibition for thromboprophylaxis, Lancet 372:6–8, 2008.
Enden T, Haig Y, Klow NE, et al: Long-term outcome after additional catheter-directed thrombolysis versus standard treatment for acute iliofemoral deep vein thrombosis (the CaVent study): a randomized controlled trial, Lancet 379:31–38, 2012.
Fischer HD, Juurlink DN, Mamdani MM, et al: Hemorrhage during warfarin therapy associated with cotrimoxazole and other urinary tract anti-infective agents, Arch Intern Med 170:617–621, 2010.
Gaballah M, Shi J, Kukreja K, et al: Endovascular thrombolysis in the management of iliofemoral thrombosis in children: a multi-institutional experience, J Vasc Interv Radiol 27:524–530, 2016.
Gage BF, Bass AR, Lin H, et al: Effect of genotype-guided warfarin dosing on clinical events and anticoagulation control among patients undergoing hip or knee arthroplasty, JAMA 318:1115–1124, 2017.
Giannakopoulos B, Krilis SA: The pathogenesis of the antiphospholipid syndrome, N Engl J Med 368:1033–1042, 2013.
Giordano P, Tesse R, Lassandro G, et al: Clinical and laboratory characteristics of children positive for antiphospholipid antibodies, Blood Transfus 10:296–301, 2012.
Goldenberg NA: Long-term outcomes of venous thrombosis in children, Curr Opin Hematol 12:370–376, 2005.
Goldenberg NA, Durham JD, Knapp-Clevenger R, et al: A thrombolytic regimen for high-risk deep venous thrombosis may substantially reduce the risk of postthrombotic syndrome in children, Blood 110:45–53, 2007.
Goldenberg NA, Pounder E, Knapp-Clevenger R, et al: Validation of upper extremity post-thrombotic syndrome outcome measurement in children, J Pediatr 157:852–855, 2010.
Gupta A, Johnson DH, Nagalla S: Antiphospholipid antibodies, JAMA 318:959–960, 2017.
The Hokusai-VTE Investigators: Edoxaban versus warfarin for the treatment of symptomatic venous thromboembolism, N Engl J Med 369:1406–1414, 2013.
Kearon C, Akl EA, Ornelas J, et al: Antithrombotic therapy for VTE disease: CHEST guideline and expert panel report, Chest 149:315–352, 2016.
Kuhle S, Eulmesekian P, Massicotte P, et al: A clinically significant incidence of bleeding in critically ill children receiving therapeutic doses of unfractionated heparin: a prospective cohort study, Haematologica 92:244–247, 2007.
Kyrle PA: Vitamin K antagonists: self-determination by self-monitoring?, Lancet 379:292–293, 2012.
Landenfeld CS: Noninvasive diagnosis of deep vein thrombosis, JAMA 300:1696–1697, 2008.
Le Gal G, Kercret G, Yahmed KB, et al: Diagnostic value of single compete compression ultrasonography in pregnant and postpartum women with suspected deep vein thrombosis: prospective study, BMJ 344:16, 2012.
Lee A, Crowther M: Practical issues with vitamin K antagonists: elevated INRs, low time-in-therapeutic range, and warfarin failure, J Thromb Thrombolysis 31:249–258, 2011.
Lyle CA, Sidonio RF, Goldenberg NA: New developments in pediatric venous thromboembolism and anticoagulation, including the target-specific oral anticoagulants, Curr Opin Pediatr 27:18–25, 2015.
Manco-Johnson MJ: How I treat venous thrombosis in children, Blood 107:21–29, 2007.
Manco-Johnson MJ, Wang M, Goldenberg NA, et al: Treatment, survival, and thromboembolic outcomes of thrombotic storm in children, J Pediatr 161:682–688, 2012.
The Medical Letter: Antithrombotic drugs, Med Lett 56:103–108, 2014.
The Medical Letter: Dabigatran etexilate (Pradaxa) — a new oral anticoagulant, Med Lett Drugs Ther 52(1351):89–90, 2010.
The Medical Letter: Prothrombin complex concentrates to reverse warfarin-related bleeding, Med Lett Drugs Ther 53:78–80, 2011.
The Medical Letter: Rethinking warfarin for atrial fibrillation, Med Lett Drugs Ther 55:77–78, 2013.
The Medical Letter: Apixaban (Eliquis) — a new oral anticoagulant for atrial fibrillation, Med Lett Drugs Ther 55:9–10, 2013.
The Medical Letter: Kcentra: a 4-factor prothrombin complex concentrate for reversal of warfarin anticoagulation, Med Lett Drugs Ther 55:53, 2013.
The Medical Letter: Edoxaban (Savaysa): the fourth new oral anticoagulant, Med Lett 57:43–46, 2015.
Miyawaki Y, Suzuki A, Fujita J, et al: Thrombosis from a prothrombin mutation conveying antithrombin resistance, N Engl J Med 366:2390–2396, 2012.
Monagle P, Chan A, Goldenberg N, et al: Antithrombotic therapy in neonates and children: American college of chest physicians Evidence-based clinical practice guidelines, Chest 141:e737S–801S, 2012.
Pollack CV Jr, Reilly PA, van Ryn J, et al: Idarucizumab for dabigatran reversal: full cohort analysis, N Engl J Med 377:431–441, 2017
Raffini L, Huang YS, Witmer C, et al: Dramatic increase in venous thromboembolism in children’s hospitals in the United States from 2001 to 2007, Pediatrics 124:1001–1008, 2009.
Richard AA, Kim S, Moffett BS, et al: Comparison of anti-xa levels in obese and non-obese pediatric patients receiving treatment doses of enoxaparin, J Pediatr 162:293–296, 2013.
Riley TR, Gauthier-Lewis ML, Sanchez CK, Douglas JS: Role of agents for reversing the effects of target-specific oral anticoagulants, Am J Health Syst Pharm 74:54–61, 2017.
Rondina MT, Pendleton RC, Wheeler M, et al: The treatment of venous thromboembolism in special populations, Thromb Res 119:391–402, 2007.
Roy PM: Diagnosis of venous thromboembolism, BMJ 339:412–413, 2009.
Ruiz-Irastorza G, Crowther M, Branch W, et al: Antiphospholipid syndrome, Lancet 376:1498–1506, 2010.
Saracco P, Bagna R, Gentilomo C, et al: Clinical data of neonatal systemic thrombosis, J Pediatr 171:60–66, 2016.
Schulman S, Kearon C, Kakkar AJ, et al: Dabigatran versus warfarin in the treatment of acute venous thromboembolism, N Engl J Med 361:2342–2352, 2009.
Schulman S, Kearon C, Kakkar AK, et al: Extended use of dabigatran, warfarin, or placebo in venous thromboembolism, N Engl J Med 368:709–718, 2013.
Siegel DM, Curnutte JT, Connolly SJ, et al: Andexanet alfa for the reversal of factor xa inhibitor activity, N Engl J Med 373:2413–2424, 2015.
Stegeman BH, de Bastos M, Rosendaal FR, et al: Different combined oral contraceptives and the risk of venous thrombosis: systematic review and network meta-analysis, BMJ 347:11, 2013.
Tremor CC III, Chung RJ, Michelson AD, et al: Hormonal contraception and thrombotic risk: a multidisciplinary approach, Pediatrics 127:347–357, 2011.
Tuckuviene R, Christensen AL, Helgestad J, et al: Pediatric venous and arterial noncerebral thromboembolism in Denmark: a nationwide population-based study, J Pediatr 159:663–669, 2011.
Van Es N, Coppens M, Schulman S, et al: Direct oral anticoagulants compared with vitamin K antagonists for acute venous thromboembolism: evidence from phase 3 trials, Blood 124:1968–1975, 2014.
Verheugt FWA, Granger CB: Oral anticoagulants for stroke prevention in atrial fibrillation: current status, special situations, and unmet needs, Lancet 386:303–310, 2015.
Young G, Albisetti M, Bonduel M, et al: Impact of inherited thrombophilia on venous thromboembolism in children: a systematic review and meta-analysis of observational studies, Circulation 118:1373–1382, 2008.
Burke CW: Vitamin K deficiency bleeding: overview and considerations, J Pediatr Health Care 27:215–221, 2013.
Pipe SW, Goldenberg NA, et al: Acquired disorders of hemostasis. In Orkin SH, Fisher DE, Ginsberg D, editors: Nathan and Oski’s hematology of infancy and childhood, ed 8, Philadelphia, 2015, Saunders Elsevier, pp 1103–1124.
Yates SR, Sarode R: New strategies for effective treatment of vitamin K antagonist– associated bleeding, J Thromb Haemost 13(Suppl 1):S180–S186, 2015.
Magnusson M, Ignjatovic V, Hardikar W, Monagle P: A conceptual and practical approach to haemostasis in paediatric liver disease, Arch Dis Child 101:854–859, 2016.
Pandit TN, Sarode R: Blood component support in acquired coagulopathic conditions: is there a method to the madness?, Am J Hematol 87(Suppl 1):S56–S62, 2012.
Pipe SW, Goldenberg NA: Acquired disorders of hemostasis. In Orkin SH, Fisher DE, Ginsberg D, et al, editors: Nathan and Oski’s hematology of infancy and childhood, ed 8, Philadelphia, 2015, Saunders Elsevier, pp 1103–1124.
Tripodi A, Mannucci PM: The coagulopathy of chronic liver disease, N Engl J Med 365(2):147–156, 2011.
Wicklund BM: Bleeding and clotting disorders in pediatric liver disease, Hematology Am Soc Hematol Educ Program 2011:170–177, 2011.
Collins WP, Chalmers E, Hart D, et al: Diagnosis and management of acquired coagulation inhibitors: a guideline from UKHCDO, Br J Haematol 162:758–773, 2013.
Janbain M, Leissinger CA, Kruse-Jarres R: Acquired hemophilia a: emerging treatment options, J Blood Med 8:143–150, 2015.
Kessler CM, Kn?bl P: Acquired hemophilia: an overview for clinical practice, Eur J Haematol 95(Suppl 81):36–44, 2015.
Mazodier K, Arnaud L, Mathian A, et al: Lupus anticoagulant-hypoprothrombinemia syndrome: report of 8 cases and review of the literature, Medicine (Baltimore) 91(5):251–260, 2012.
Negrini S, Pappalardo F, Murdaca G, et al: The antiphospholipid antibody syndrome: from pathophysiology to treatment, Clin Exp Med 17(3):257–267, 2017.
Pipe SW, Goldenberg NA: Acquired disorders of hemostasis. In Orkin SH, Fisher DE, Ginsberg D, et al, editors: Nathan and Oski’s hematology of infancy and childhood, ed 8, Philadelphia, 2015, Saunders Elsevier, pp 1103–1124.
Hook KM, Abrams CS: The loss of homeostasis in hemostasis: new approaches in treating and understanding acute disseminated intravascular coagulation in critically ill patients, Clin Transl Sci 5(1):85–92, 2012.
Levi M, Toh CH, Thachil J, et al: Guidelines for the diagnosis and management of disseminated intravascular coagulation. British committee for standards in haematology, Br J Haematol 145:24–33, 2009.
Pipe SW, Goldenberg NA: Acquired disorders of hemostasis. In Orkin SH, Fisher DE, Ginsberg D, et al, editors: Nathan and Oski’s hematology of infancy and childhood, ed 8, Philadelphia, 2015, Saunders Elsevier, pp 1103–1124.
Thachil J, Toh CH: Current concepts in the management of disseminated intravascular coagulation, Thromb Res 129(Suppl 1):S54–S59, 2012.
Boyle S, White RH, Brunson A, et al: Splenectomy and the incidence of venous thromboembolism and sepsis in patients with immune thrombocytopenia, Blood 121(23):4782–4790, 2013.
Chaturvedi S, McCrae KR: Treatment of chronic immune thrombocytopenia in children with romiplostim, Lancet 388:4–6, 2016.
Cooper N, Bussel JB: The long-term impact of rituximab for childhood immune thrombocytopenia, Curr Rheumatol Rep 12(2):94–100, 2010.
Heitink-Poll? KM, Njisten J, Boonacker CW, et al: Clinical and laboratory predictors of chronic immune thrombocytopenia in children: a systematic review and metaanalysis, Blood 124:3295–3307, 2015.
Kohne T, Imbach P: Management of children and adolescents with primary immune thrombocytopenia: controversies and solutions, Vox Sang 104(1):55–66, 2013.
Neunert C, Noroozi N, Norman G, et al: Severe bleeding events in adults and children with primary immune thrombocytopenia: a systematic review, J Thromb Haemost 13:457–464, 2015.
Neunert CE, Buchanan GR, Imbach P, et al: Bleeding manifestations and management of children with persistent and chronic immune thrombocytopenia: data from the intercontinental cooperative ITP Study Group (ICIS), Blood 121(22):4457–4462, 2013.
Neunert C, Lim W, Crowther M, et al: American society of hematology. The American society of hematology 2011 evidence-based practice guideline for immune thrombocytopenia, Blood 117(16):4190–4207, 2011.
O’Leary ST, Glanz JM, McClure DL, et al: The risk of immune thrombocytopenic purpura after vaccination in children and adolescents, Pediatrics 129(2):248–255, 2012.
Price V: Auto-immune lymphoproliferative disorder and other secondary immune thrombocytopenias in childhood, Pediatr Blood Cancer 60(Suppl 1):S12–S14, 2013.
Psaila B, Cooper N: B-cell depletion in immune thrombocytopenia, Lancet 385:1599–1600, 2015.
Psaila B, Petrovic A, Page LK, et al: Intracranial hemorrhage (ICH) in children with immune thrombocytopenic (ITP): study of 40 cases, Blood 114:4777–4783, 2009.
Revel-Vilk S, Yacobovich J, Frank S, et al: Age and duration of bleeding symptoms at diagnosis best predict resolution of childhood immune thrombocytopenia at 3,6, and 12 months, J Pediatr 163:1335–1339, 2013.
Teachey DT, Lambert MP: Diagnosis and management of autoimmune cytopenias in childhood, Pediatr Clin North Am 60:1489–1511, 2013.
Yacobovich J, Revel-Vilk S, Tamary H: Childhood immune thrombocytopenia — who will spontaneously recover?, Semin Hematol 50(Suppl 1):S71–S74, 2013.
Cuker A: Management of the multiple phases of heparin-induced thrombocytopenia, Thromb Haemost 116:835–842, 2016.
Mariotte E, Veyradier A: Thrombotic thrombocytopenic purpura: from diagnosis to therapy, Curr Opin Crit Care 21:593–601, 2015.
Ortel TL, Erkan D, Kitchens CS: How I treat catastrophic thrombotic syndromes, Blood 126:1285–1293, 2015.
Peyvandi F, Scully M, Kremer-Hovinga JA, et al: Caplacizumab for acquired thrombotic thrombocytopenia purpura, N Engl J Med 374(6):511–522, 2016.
Reese JA, Muthurajah DS, Kremer Hovinga JA, et al: Children and adults with thrombotic thrombocytopenic purpura associated with severe, acquired ADAMTS13 deficiency: comparison of incidence, demographic and clinical features, Pediatr Blood Cancer 60:1676–1682, 2013.
Sadler JE, Moake JL, Miyata T, George JN: Recent advances in thrombotic thrombocytopenic purpura, Hematology Am Soc Hematol Educ Program 2004:407–423, 2004.
Scully M, Cataland S, Coppo P, et al: Consensus on the standardization of terminology in thrombotic thrombocytopenic purpura and related thrombotic microangiopathies, J Thromb Haemost 15:312–322, 2016.
Croteau SE, Gupta D: The clinical spectrum of kaposiform hemangioendothelioma and tufted angioma, Semin Cutan Med Surg 35:147–152, 2016.
Drolet BA, Trenor CC 3rd, Brand?o LR, et al: Consensus-derived practice standards plan for complicated kaposiform hemangioendothelioma, J Pediatr 163:285–291, 2013.
Albers CA, Newbury-Ecob R, Ouwehand WH, Ghevaert C: New insights into the genetic basis of TAR (thrombocytopenia-absent radii) syndrome, Curr Opin Genet Dev 23:316–323, 2013.
Balduini CL, Savoia A: Genetics of familial forms of thrombocytopenia, Hum Genet 131(12):1821–1832, 2012.
Geddes AE: Congenital amegakaryocytic thrombocytopenia, Pediatr Blood Cancer 57:199–203, 2011.
Worth AJ, Thrasher AJ: Current and emerging treatment options for Wiskott-aldrich syndrome, Expert Rev Clin Immunol 11:1015–1032, 2015.
Curtis BR: Recent progress in understanding the pathogenesis of fetal and neonatal alloimmune thrombocytopenia, Br J Haematol 171:671–682, 2015.
Peterson JA, McFarland JG, Curtis BR, et al: Neonatal alloimmune thrombocytopenia: pathogenesis, diagnosis and management, Br J Haematol 161(1):3–14, 2013.
Mitchell O, Feldman DM, Diakow M, Sigal SH: The pathophysiology of thrombocytopenia in chronic liver disease, Hepat Med 8:39–50, 2016.
Blavignac J, Bunimov N, Rivard GE, Hayward CP: Quebec platelet disorder: update on pathogenesis, diagnosis, and treatment, Semin Thromb Hemost 37:713–720, 2011.
Gunay-Aygun M, Huizing M, Gahl WA: Molecular defects that affect platelet dense granules, Semin Thromb Hemost 30(5):537–547, 2004.
Gunay-Aygun M, Falik-Zaccai TC, Vilboux T, et al: NBEAL2 is mutated in gray platelet syndrome and is required for biogenesis of platelet ?-granules, Nat Genet 43:732–734, 2011.
Hinckley J, Di Paola J: Genetic basis of congenital platelet disorders, Hematology Am Soc Hematol Educ Program 2014:337–342, 2014.
Lambert MP, Poncz M: Inherited platelet disorders. In Orkin SH, Fisher DE, Ginsberg D, et al, editors: Nathan and Oski’s hematology of infancy and childhood, ed 8, Philadelphia, 2015, Saunders Elsevier, pp 1010–1027.
Lambert MP: Update on the inherited platelet disorders, Curr Opin Hematol 22:460–466, 2015.
Loredana Asztalos M, Schafernak KT, Gray J, et al: Hermansky-pudlak syndrome: report of two patients with updated genetic classification and management recommendations, Pediatr Dermatol 34(6):638–646, 2017.
Monteferrario D, Bolar NA, Marneth AE, et al: A dominant-negative GFI1B mutation in the gray platelet syndrome, N Engl J Med 370(3):245–252, 2014.
Nurden AT, Nurden P: Congenital platelet disorders and understanding of platelet function, Br J Haematol 165:165–178, 2014.
Quiroga T, Mezzano D: Is my patient a bleeder? A diagnostic framework for mild bleeding disorders, Hematology Am Soc Hematol Educ Program 2012:466–474, 2012.
Seward SL Jr, Gahl WA: Hermansky-pudlak syndrome: health care throughout life, Pediatrics 132:153–160, 2013.
Amos LE, Carpenter SL, Hoeltzel MF: Lost at sea in search of a diagnosis: a case of unexplained bleeding, Pediatr Blood Cancer 63:1305–1306, 2016.
Hickey SE, Varga EA, Kerlin B: Epidemiology of bleeding symptoms and hypermobile Ehlers-danlos syndrome in paediatrics, Haemophilia 22:e490–e493, 2016.
De Porto AP, Lammers AJ, Bennink RJ, et al: Assessment of splenic function, Eur J Clin Microbiol Infect Dis 29(12):1465–1473, 2010.
Mebius RE, Kraal G: Structure and function of the spleen, Nat Rev Immunol 5(8):606–616, 2005.
Nemati M, Hajalioghli P, Jahed S, et al: Normal values of spleen length and volume: an ultrasonographic study in children, Ultrasound Med Biol 42(8):1771–1778, 2016.
Rogers ZR, Wang WC, Luo Z, et al: Biomarkers of splenic function in infants with sickle cell anemia: baseline data from the BABY HUG trial, Blood 117(9):2614–2617, 2011.
Rousan LA, Fataftah J, Al-Omari MH, et al: Sonographic assessment of liver and spleen size based on age, height, and weight: evaluation of jordanian children, Minerva Pediatr 2015. [Epub ahead of print]
Thapa NB, Shah S, Pradhan A, et al: Sonographic assessment of the normal dimensions of liver, spleen, and kidney in healthy children at tertiary care hospital, Kathmandu Univ Med J (KUMJ) 13(52):286–291, 2015.
Ancliff P, Hann I: Splenomegaly. In Sills RH, editor: Practical algorithms in pediatric hematology and oncology, Basel, 2003, Karger.
Baris HN, Cohen IJ, Mistry PK: Gaucher disease: the metabolic defect, pathophysiology, phenotypes and natural history, Pediatr Endocrinol Rev Suppl 1:72–81, 2014.
Dabritz J, Worch J, Materna U, et al: Life-threatening hypersplenism due to idiopathic portal hypertension in early childhood: case report and review of the literature, BMC Gastroenterol 10:122, 2010.
Haley K: Congenital hemolytic anemia, Med Clin North Am 101(2):361–374, 2017.
Thomas AS, Mehta AB, Hughes DA: Diagnosing gaucher disease: an on-going need for increased awareness amongst haematologists, Blood Cells Mol Dis 50(3):212–217, 2013.
US Department of Health and Human Services, Centers for Disease Control and Prevention: Splenomegaly of unknown etiology in congolese refugees applying for resettlement to the United States—Uganda, MMWR 65(35):943–944, 2015.
Weinreb NJ, Rosenbloom BE: Splenomegaly, hypersplenism, and hereditary disorders with splenomegaly, O J Gen 3:24–43, 2013
Alexakis N, Dardamanis D, Albanopoulos K, et al: Incidence, risk factors, and outcome of portal vein thrombosis after laparoscopic-assisted splenectomy in beta-thalassemia patients: a prospective exploratory study, J Laparoendosc Adv Surg Tech A 23(2):123–128, 2013.
Audia S, Santegoets K, Laarhoven AG, et al: Fc? receptor expression on splenic macrophages in adult immune thrombocytopenia, Clin Exp Immunol 188(2):275–282, 2017.
Daodu O, Brindle M, Alvarez-Allende CR, et al: Outcomes of an accelerated care pathway for pediatric blunt solid organ injuries in a public healthcare system, J Pediatr Surg 52(5):826–831, 2017.
Das A, Bansal D, Ahluwalia J, et al: Risk factors for thromboembolism and pulmonary artery hypertension following splenectomy in children with hereditary spherocytosis, Pediatr Blood Cancer 61(1):29–33, 2014.
De Porto APNA, Lammers AJJ, Bennink RJ: Assessment of splenic function, Eur J Clin Microbiol Infect Dis 29:1465–1473, 2010.
Di Sabatino A, Carsetti R, Corazza GR: Post-splenectomy and hyposplenic states, Lancet 378(9785):86–97, 2011.
Erdem SB, Genel F, Erdur B, et al: Asplenia in children with congenital heart disease as a cause of poor outcome, Cent Eur J Immunol 40(2):266–269, 2015.
Frey MK, Alias S, Winter MP, et al: Splenectomy is modifying the vascular remodeling of thrombosis, J Am Heart Assoc 3(1):e000772, 2014.
Kimberlin DW: Pneumococcal infections. In Red book: 2015 report of the committee on infectious diseases, ed 30, Elk Grove Village, IL, 2015, American Academy of Pediatrics.
Kimmig LM, Palevsky HI: Review of the association between splenectomy and chronic thromboembolic pulmonary hypertension, Ann Am Thorac Soc 13(6):945–954, 2016.
Linnaus ME, Langlais CS, Garcia NM, et al: Failure of nonoperative management of pediatric blunt liver and spleen injuries: a prospective Arizona-Texas-OklahomaMemphis-Arkansas Consortium study, J Trauma Acute Care Surg 82(4):672–679, 2017.
Nuorti JP, Whitney CG, Centers for Disease Control and Prevention (CDC): Prevention of pneumococcal disease among infants and children: use of 13-valent pneumococcal conjugate vaccine and 23-valent pneumococcal polysaccharide vaccine—recommendations of the advisory committee on immunization practices (ACIP), MMWR Recomm Rep 59:1, 2010.
Palkar AV, Agrawal A, Verma S, et al: Post splenectomy related pulmonary hypertension, World J Respirol 5(2):69–77, 2015.
Piano Mortari E, Baban A, Cantarutti N, et al: Heterotaxy syndrome with and without spleen: different infection risk and management, J Allergy Clin Immunol 139(6):1981–1984, 2017.
Rice HE, Crary SE, Langer JC, et al: Comparative effectiveness of different types of splenectomy for children with congenital hemolytic anemias, J Pediatr 160(4):684–689.e13, 2012.
Rubin LG, Schaffner W: Care of the asplenic patient, N Engl J Med 371:349–356, 2014.
Theilacker C, Ludewig K, Serr A, et al: Splenectomy, Pneumococcus, and fulminant infection SPLEEN OFF study group: overwhelming postsplenectomy infection: a prospective multicenter cohort study, Clin Infect Dis 62(7):871–878, 2016.
Klotz L, Norman S, Vieira JM: Cardiac lymphatics are heterogeneous in origin and respond to injury, Nature 522:62–67, 2015.
Louveau A, Smirnov I, Keyes TJ, et al: Structural and functional features of central nervous system lymphatic vessels, Nature 523:337–341, 2015.
Semo J, Nicenbiom J, Yaniv K: Development of the lymphatic system: new questions and paradigms, Development 143:924–935, 2016.
Yang Y, Guillermo O: Development of the mammalian vasculature, J Clin Invest 124:888–897, 2014.
Adams DM, Trenor CC III, Hammill AM, et al: Efficacy and safety of sirolimus in the treatment of complicated vascular anomalies, Pediatrics 137(2):e201153257, 2016.
Bagrodia N, Defnet AM, Kandel JJ: Management of lymphatic malformations in children, Curr Opin Pediatr 27:356–363, 2015.
Dentici ML, Di Pede A, Lepri FR, et al: Kabuki syndrome: clinical and molecular diagnosis in the first year of life, Arch Dis Child 100:158–164, 2015.
Frosk P, Chodirker B, Simard L, et al: A novel CCBE1 mutation leading to a mild form of hennekam syndrome: case report and review of the literature, BMC Med Genet 16:28, 2015.
Garcia-Montero P, del Boz J, Sanchez-Martinez M, et al: Microcystic lymphatic malformation successfully treated with topical rapamycin, Pediatrics 139(5):e20162105, 2016.
Luks VL, Kamitaki N, Vivero MP, et al: Lymphatic and other malformative/overgrowth disorders are caused by somatic mutations in PIK3CA, J Pediatr 166:1048–1054, 2015.
Trenor CC II, Chaudry G: Complex lymphatic anomalies, Semin Pediatr Surg 23:186–190, 2014.
Vlahovic AM, Vlahovic NS, Haxhija EQ: Sirolimus for the treatment of a massive capillary-lymphatico-venous malformation: a case report, Pediatrics 136(2):e513– e516, 2015.
Bozlak S, Varkal MA, Yildiz I, et al: Cervical lymphadenopathies in children: a prospective cohort study, Int J Pediatr Otorhinolaryngol 82:81–87, 2016.
Gov-Ari E, Hopewell BL: Correlation between pre-operative diagnosis and post-operative pathology reading in pediatric neck masses: a review of 281 cases, Int J Pediatr Otorhinolaryngol 79:2–7, 2015.
Lee DY, Seok J, Kim Y, et al: Neck computed tomography in pediatric neck mass as initial evaluation in ED: is it malpractice, Am J Emerg Med 32(10):1237–1240, 2014.
Locke R, Comfort R, Kubba H: When does an enlarged cervical lymph node in a child need excision? A systematic review, Int J Pediatr Otorhinolaryngol 78:393–401, 2014.
Penn EB Jr, Goudy SL: Pediatric inflammatory adenopathy, Otolaryngol Clin North Am 48:137–151, 2015.
Pynnonen MA, Gillespie MB, Roman B, et al: Clinical practice guideline: evaluation of the neck mass in adults, Otolaryngol Head Neck Surg 157(2S):S1–S30, 2017.
Cuglievan B, Miranda RN: Kikuchi-fujimoto disease, Blood 129(7):917, 2017.
Dumas G, Prendki V, Haroche J, et al: Kikuchi-fujimoto disease, Medicine (Baltimore) 93(24):372–380, 2014.
Kang HM, Kim JY, Choi EH, et al: Clinical characteristics of severe histiocytic necrotizing lymphadenitis (kikuchi-fujimoto disease) in children, J Pediatr 171:208–212, 2016.
Kim JY, Lee H, Yun BL: Ultrasonographic findings of kikuchi cervical lymphadenopathy in children, Ultrasonography 36(1):66–70, 2017.
Cooper SL, Arceci RJ, Gamper CJ, et al: Successful treatment of recurrent autoimmune cytopenias in the context of sinus histiocytosis with massive lymphadenopathy using sirolimus, Pediatr Blood Cancer 63:358–360, 2016.
Emile JF, Abla O, Fraitag S, et al: Revised classification of histiocytoses and neoplasms of the macrophage–dendritic cell lineages, Blood 127(22):2672–2681, 2016.
Haroche J, Abla O: Uncommon histiocytic disorders: Rosai-dorfman, juvenile xanthogranuloma, and Erdheim-chester disease, Hematology Am Soc Hematol Educ Program 2015:571–578, 2015.
Simko SJ, Tran HD, Jones J, et al: Clofarabine salvage therapy in refractory multifocal histiocytic disorders, including langerhans cell histiocytosis, juvenile xanthogranuloma and Rosai-dorfman disease, Pediatr Blood Cancer 61:479–487, 2014.
Fajgenbaum DC, Uldrick TS, Bagg A, et al: International, evidence-based consensus diagnostic criteria for HHV-8-negative/idiopathic multicentric castleman disease, Blood 129(12):1646–1657, 2017.
Fajgenbaum DC, van Rhee F, Nabel CS: HHV-8-negative, idiopathic multicentric castleman disease: novel insights into biology, pathogenesis, and therapy, Blood 123(19):2924–2933, 2014.
Jain P, Verstovsek S, Loghavi S, et al: Durable remission with rituximab in a patient with an unusual variant of Castleman’s disease with myelofibrosis: TAFRO syndrome, Am J Hematol 90(11):1091–1092, 2015.
Liu AY, Nabel CS, Finkelman BS, et al: Idiopathic multicentric Castleman’s disease: a systematic literature review, Lancet Hematol 3(4):e163–e175, 2016.
Sbihi Z, Dossier A, Boutboul D, et al: INKT and memory B-cell alterations in HHV-8 multicentric castleman disease, Blood 129(7):855–865, 2017.
Smith C, Lee-Miller C, Dishop MK, et al: Multicentric castleman disease presenting with fever, J Pediatr 165:1261–1265, 2014.
Turcotte LM, Correll CK, Reed RC, et al: Sustained remission of severe multicentric castleman disease following multiagent chemotherapy and tocilizumab maintenance, Pediatr Blood Cancer 61:737–739, 2014.