Photo of Ellis J. Neufeld,  MD, PhD

Ellis J. Neufeld, MD, PhD

Boston Children's Hospital

Boston Children's Hospital
Phone: (617) 919-2139
Fax: (617) 730-0934

Ellis J. Neufeld, MD, PhD

Boston Children's Hospital


  • Egan Family Foundation Professor of Transitional Medicine in the Department of Pediatrics, Pediatrics, Harvard Medical School
  • Associate Chief, Hematology/Oncology, Boston Children's Hospital
  • Director, Boston Hemophilia Center, Hematology, Brigham And Women's Hospital
  • Co-Chief, Clinical Research Center, Boston Children's Hospital
  • Co-chair, Harvard Catalyst Child Health Program, Harvard Medical School


Research Abstract

Dr. Neufeld studies genetic blood disorders, iron overload due to transfusions, and autoimmune blood diseases. These are non-malignant conditions.

His laboratory work has focused on candidate genes for inherited anemias.

Clinical research interests include ITP (Immune Thrombocytopenia, a disorder which causes destruction of blood platelets), Thalassemia, transfusional iron overload, and genetic disorders of blood clotting, including hemophilia, platelet disorders, and thrombotic disorders.


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  • Schoettler M, Elisofon SA, Kim HB, Blume ED, Rodig N, Boyer D, Neufeld EJ, Grace RF. Treatment and outcomes of immune cytopenias following solid organ transplant in children. Pediatr Blood Cancer 2014. PubMed
  • Vichinsky E, Neumayr L, Trimble S, Giardina PJ, Cohen AR, Coates T, Boudreaux J, Neufeld EJ, Kenney K, Grant A, Thompson AA, . Transfusion complications in thalassemia patients: a report from the Centers for Disease Control and Prevention (CME). Transfusion 2014; 54:972-81. PubMed
  • Trachtenberg FL, Gerstenberger E, Xu Y, Mednick L, Sobota A, Ware H, Thompson AA, Neufeld EJ, Yamashita R. Relationship among chelator adherence, change in chelators, and quality of life in Thalassemia. Qual Life Res 2014. PubMed
  • Morrison JF, Neufeld EJ, Grace RF. The use of erythropoietin-stimulating agents versus supportive care in newborns with hereditary spherocytosis: a single centre's experience. Eur J Haematol 2014. PubMed
  • Nichols-Vinueza DX, White MT, Powell AJ, Banka P, Neufeld EJ. MRI guided iron assessment and oral chelator use improve iron status in thalassemia major patients. Am J Hematol 2014. PubMed
  • Klaassen RJ, Barrowman N, Merelles-Pulcini M, Vichinsky EP, Sweeters N, Kirby-Allen M, Neufeld EJ, Kwiatkowski JL, Wu J, Vickars L, Blanchette VS, Forgie M, Yamashita R, Wong-Rieger D, Young NL. Validation and reliability of a disease-specific quality of life measure (the TranQol) in adults and children with thalassaemia major. British Journal of Haematology 2014; 164:431-7. PubMed
  • Croteau SE, Luo HY, Lehmann LE, Chui DH, Neufeld EJ. Novel dominant 硫-thalassemia: Hb Boston-Kuwait [codon 139/140(+T)]. Pediatr Blood Cancer 2013; 60:E131-4. PubMed
  • Thompson AA, Kim HY, Singer ST, Vichinsky E, Eile J, Yamashita R, Giardina PJ, Olivieri N, Parmar N, Trachtenberg F, Neufeld EJ, Kwiatkowski JL, . Pregnancy outcomes in women with thalassemia in North America and the United Kingdom. Am J Hematol 2013; 88:771-3. PubMed
  • Morris CR, Kim HY, Wood JC, Porter JB, Klings ES, Trachtenberg FL, Sweeters N, Olivieri NF, Kwiatkowski JL, Virzi L, Singer ST, Taher A, Neufeld EJ, Thompson AA, Sachdev V, Larkin S, Suh JH, Kuypers FA, Vichinsky EP. Sildenafil therapy in thalassemia patients with doppler-defined risk for pulmonary hypertension. Haematologica 2013. PubMed
  • Switzer WM, Shankar A, Trimble SR, Thompson AA, Giardina PJ, Cohen AR, Coates TD, Vichinsky E, Neufeld EJ, Boudreaux JM, Heneine W. Human T Cell Lymphotropic Virus Type 1 Infection Among U.S. Thalassemia Patients. AIDS Res Hum Retroviruses 2013. PubMed
  • Cherry AB, Gagne KE, McLoughlin EM, Baccei A, Gorman B, Hartung O, Miller JD, Zhang J, Zon RL, Ince TA, Neufeld EJ, Lerou PH, Fleming MD, Daley GQ, Agarwal S. Induced Pluripotent Stem Cells with a Pathological Mitochondrial DNA Deletion. Stem Cells 2013. PubMed
  • Walter PB, Porter J, Evans P, Kwiatkowski JL, Neufeld EJ, Coates T, Giardina PJ, Grady RW, Vichinsky E, Olivieri N, Trachtenberg F, Alberti D, Fung E, Ames B, Higa A, Harmatz P, . Increased leucocyte apoptosis in transfused 硫-thalassaemia patients. British Journal of Haematology 2013; 160:399-403. PubMed
  • Neufeld EJ, Saxena K, Kessler CM, Cooper DL, . Dosing, efficacy, and safety of recombinant factor VIIa (rFVIIa) in pediatric versus adult patients: The experience of the Hemostasis and Thrombosis Research Society (HTRS) Registry (2004-2008). Pediatr Blood Cancer 2013. PubMed
  • Matzdorff A, Neufeld EJ, Roganovic J. To treat or not to treat--from guidelines to individualized patient management. Semin Hematol 2013; 50 Suppl 1:S12-7. PubMed
  • Armand P, Sainvil MM, Kim HT, Rhodes J, Cutler C, Ho VT, Koreth J, Alyea EP, Neufeld EJ, Kwong RY, Soiffer RJ, Antin JH. Does iron overload really matter in stem cell transplantation? Am J Hematol 2012. PubMed
  • Morris CR, Kim HY, Trachtenberg F, Wood J, Quinn CT, Sweeters N, Kwiatkowski JL, Thompson AA, Giardina PJ, Boudreaux J, Olivieri NF, Porter JB, Neufeld EJ, Vichinsky EP, . Risk factors and mortality associated with an elevated tricuspid regurgitant jet velocity measured by Doppler-echocardiography in thalassemia: a Thalassemia Clinical Research Network report. Blood 2011; 118:3794-3802. PubMed
  • Sobota A, Graham DA, Neufeld EJ, Heeney MM. Thirty-day readmission rates following hospitalization for pediatric sickle cell crisis at freestanding children's hospitals: Risk factors and hospital variation. Pediatr Blood Cancer 2011. PubMed
  • Grace RF, Bennett CM, Ritchey AK, Jeng M, Thornburg CD, Lambert MP, Neier M, Recht M, Kumar M, Blanchette V, Klaassen RJ, Buchanan GR, Kurth MH, Nugent DJ, Thompson AA, Stine K, Kalish LA, Neufeld EJ. Response to steroids predicts response to rituximab in pediatric chronic immune thrombocytopenia. Pediatr Blood Cancer 2011. PubMed
  • Grace RF, Long M, Kalish LA, Neufeld EJ. Applicability of 2009 international consensus terminology and criteria for immune thrombocytopenia to a clinical pediatric population. Pediatr Blood Cancer 2011. PubMed
  • Sobota A, Neufeld EJ, Sprinz P, Heeney MM. Transition from pediatric to adult care for sickle cell disease: results of a survey of pediatric providers. Am J Hematol 2011; 86:512-5. PubMed
  • Trachtenberg F, Vichinsky E, Haines D, Pakbaz Z, Mednick L, Sobota A, Kwiatkowski J, Thompson AA, Porter J, Coates T, Giardina PJ, Olivieri N, Yamashita R, Neufeld EJ, . Iron chelation adherence to deferoxamine and deferasirox in thalassemia. Am J Hematol 2011; 86:433-6. PubMed
  • Rienhoff HY, Viprakasit V, Tay L, Harmatz P, Vichinsky E, Chirnomas D, Kwiatkowski JL, Tapper A, Kramer W, Porter JB, Neufeld EJ. A phase 1 dose-escalation study: safety, tolerability, and pharmacokinetics of FBS0701, a novel oral iron chelator for the treatment of transfusional iron overload. Haematologica 2011; 96:521-5. PubMed
  • Quinn CT, Johnson VL, Kim HY, Trachtenberg F, Vogiatzi MG, Kwiatkowski JL, Neufeld EJ, Fung E, Oliveri N, Kirby M, Giardina PJ, . Renal dysfunction in patients with thalassaemia. British Journal of Haematology 2011; 153:111-117. PubMed
  • Thompson AA, Cunningham MJ, Singer ST, Neufeld EJ, Vichinsky E, Yamashita R, Giardina P, Kim HY, Trachtenberg F, Kwiatkowski JL, . Red cell alloimmunization in a diverse population of transfused patients with thalassaemia. British Journal of Haematology 2011; 153:121-128. PubMed
  • Trenor CC, Chung RJ, Michelson AD, Neufeld EJ, Gordon CM, Laufer MR, Emans SJ. Hormonal contraception and thrombotic risk: a multidisciplinary approach. Pediatrics 2011; 127:347-57. PubMed
  • Grace RF, Dahlberg SE, Neuberg D, Sallan SE, Connors JM, Neufeld EJ, Deangelo DJ, Silverman LB. The frequency and management of asparaginase-related thrombosis in paediatric and adult patients with acute lymphoblastic leukaemia treated on Dana-Farber Cancer Institute consortium protocols. British Journal of Haematology 2011; 152:452-9. PubMed
  • Mednick L, Yu S, Trachtenberg F, Xu Y, Kleinert DA, Giardina PJ, Kwiatkowski JL, Foote D, Thayalasuthan V, Porter JB, Thompson AA, Schilling L, Quinn CT, Neufeld EJ, Yamashita R, . Symptoms of depression and anxiety in patients with thalassemia: Prevalence and correlates in the thalassemia longitudinal cohort. Am J Hematol 2010; 85:802-5. PubMed
  • Pakbaz Z, Treadwell M, Kim HY, Trachtenberg F, Parmar N, Kwiatkowski JL, Cunningham MJ, Martin M, Sweeters N, Neufeld EJ, Giardina PJ, Olivieri N, Yamashita RC, Vichinsky E. Education and employment status of children and adults with thalassemia in North America. Pediatr Blood Cancer 2010; 55:678-83. PubMed
  • Armand P, Kim HT, Rhodes J, Sainvil MM, Cutler C, Ho VT, Koreth J, Alyea EP, Hearsey D, Neufeld EJ, Fleming MD, Steen H, Anderson D, Kwong RY, Soiffer RJ, Antin JH. Iron Overload in Patients with Acute Leukemia or MDS Undergoing Myeloablative Stem Cell Transplantation. Biol Blood Marrow Transplant 2010. PubMed
  • Vichinsky EP, Neufeld EJ. Preface to Cooley's Anemia: Ninth Symposium. Ann N Y Acad Sci 2010; 1202:ix-x. PubMed
  • Bergmann AK, Grace RF, Neufeld EJ. Genetic studies in pediatric ITP: outlook, feasibility, and requirements. Ann. Hematol. 2010; 89:S95-103. PubMed
  • Trachtenberg F, Foote D, Martin M, Carson S, Coates T, Beams O, Vega O, Merelles-Pulcini M, Giardina PJ, Kleinert DA, Kwiatkowski J, Thompson AA, Neufeld EJ, Schilling L, Thayalasuthan V, Pakbaz Z, Yamashita R, . Pain as an emergent issue in thalassemia. Am J Hematol 2010; 85:367-70. PubMed
  • Zehnder JL, Semple JW, Imbach P, Neufeld EJ, Buchanan GR, Cines DB, . Future research in ITP: an ICIS consensus. Ann. Hematol. 2010. PubMed
  • Merrell SC, Rahbar R, Alomari AI, Padua HM, Vargas SO, Neufeld EJ, Dearden JL, Mulliken JB, Greene AK. Infantile Myofibroma or Lymphatic Malformation: Differential Diagnosis of Neonatal Cystic Cervicofacial Lesions. J Craniofac Surg 2010; 21:422-6. PubMed
  • Bergmann AK, Campagna DR, McLoughlin EM, Agarwal S, Fleming MD, Bottomley SS, Neufeld EJ. Systematic molecular genetic analysis of congenital sideroblastic anemia: evidence for genetic heterogeneity and identification of novel mutations. Pediatr Blood Cancer 2009; 54:273-8. PubMed
  • Sobota A, Graham DA, Heeney MM, Neufeld EJ. Corticosteroids for acute chest syndrome in children with sickle cell disease: variation in use and association with length of stay and readmission. Am J Hematol 2009; 85:24-8. PubMed
  • Borzutzky A, Crompton B, Bergmann AK, Giliani S, Baxi S, Martin M, Neufeld EJ, Notarangelo LD. Reversible severe combined immunodeficiency phenotype secondary to a mutation of the proton-coupled folate transporter. Clin Immunol 2009; 133:287-94. PubMed
  • Bergmann AK, Sahai I, Falcone JF, Fleming J, Bagg A, Borgna-Pignati C, Casey R, Fabris L, Hexner E, Mathews L, Ribeiro ML, Wierenga KJ, Neufeld EJ. Thiamine-responsive megaloblastic anemia: identification of novel compound heterozygotes and mutation update. J Pediatr 2009; 155:888-892.e1. PubMed
  • Bergmann AK, Tamary H, Neufeld EJ. Remission from transfusion-dependence in a patient with congenital dyserythropoietic anemia (CDA) and increased intensity of iron chelation. Pediatr Blood Cancer 2009; 53:1167-8. PubMed
  • Chirnomas D, Smith AL, Braunstein J, Finkelstein Y, Pereira L, Bergmann AK, Grant FD, Paley C, Shannon M, Neufeld EJ. Deferasirox pharmacokinetics in patients with adequate versus inadequate response. Blood 2009; 114:4009-13. PubMed
  • Grace RF, Mednick RE, Neufeld EJ. Compliance with immunizations in splenectomized individuals with hereditary spherocytosis. Pediatr Blood Cancer 2009; 52:865-7. PubMed
  • Odegard KC,Zurakowski D,DiNardo JA,Castro RA,McGowan FX Jr,Neufeld EJ,Laussen PC. Prospective longitudinal study of coagulation profiles in children with hypoplastic left heart syndrome from stage I through Fontan completion. J Thorac Cardiovasc Surg 2009; 137:934-41. PubMed
  • Mueller BU,Bennett CM,Feldman HA,Bussel JB,Abshire TC,Moore TB,Sawaf H,Loh ML,Rogers ZR,Glader BE,McCarthy MC,Mahoney DH,Olson TA,Feig SA,Lorenzana AN,Mentzer WC,Buchanan GR,Neufeld EJ. One year follow-up of children and adolescents with chronic immune thrombocytopenic purpura (ITP) treated with rituximab. Pediatr Blood Cancer 2008; 52:259-62. PubMed
  • Sobota A,Neufeld EJ,Lapsia S,Bennett CM. Response to mercaptopurine for refractory autoimmune cytopenias in children. Pediatr Blood Cancer 2008; 52:80-4. PubMed
  • Neunert CE,Buchanan GR,Imbach P,Bolton-Maggs PH,Bennett CM,Neufeld EJ,Vesely SK,Adix L,Blanchette VS,Kuhne T. Severe hemorrhage in children with newly diagnosed immune thrombocytopenic purpura. Blood 2008; 112:4003-8. PubMed
  • Deborah Chirnomas S,Geukes-Foppen M,Barry K,Braunstein J,Kalish LA,Neufeld EJ,Powell AJ. Practical implications of liver and heart iron load assessment by T2*-MRI in children and adults with transfusion-dependent anemias. Am J Hematol 2008; 83:781-3. PubMed
  • Abel GA, Neufeld EJ, Sorel M, Weeks JC. Direct-to-consumer advertising for bleeding disorders: a content analysis and expert evaluation of advertising claims. J Thromb Haemost 2008; 6:1680-4. PubMed
  • Bennett CM,Boye E,Neufeld EJ. Female monozygotic twins discordant for hemophilia A due to nonrandom X-chromosome inactivation. Am J Hematol 2008; 83:778-80. PubMed
  • Harmatz P,Jonas MM,Kwiatkowski JL,Wright EC,Fischer R,Vichinsky E,Giardina PJ,Neufeld EJ,Porter J,Olivieri N. Safety and efficacy of pegylated interferon alpha-2a and ribavirin for the treatment of hepatitis C in patients with thalassemia. Haematologica 2008; 93:1247-51. PubMed
  • Mullen MP,Wessel DL,Thomas KC,Gauvreau K,Neufeld EJ,McGowan FX Jr,Dinardo JA. The incidence and implications of anti-heparin-platelet factor 4 antibody formation in a pediatric cardiac surgical population. Anesth Analg 2008; 107:371-8. PubMed
  • Walter PB,Macklin EA,Porter J,Evans P,Kwiatkowski JL,Neufeld EJ,Coates T,Giardina PJ,Vichinsky E,Olivieri N,Alberti D,Holland J,Harmatz P. Inflammation and oxidant-stress in beta-thalassemia patients treated with iron chelators deferasirox (ICL670) or deferoxamine: an ancillary study of the Novartis CICL670A0107 trial. Haematologica 2008; 93:817-25. PubMed
  • Porter J, Galanello R, Saglio G, Neufeld EJ, Vichinsky E, Cappellini MD, Olivieri N, Piga A, Cunningham MJ, Soulieres D, Gattermann N, Tchernia G, Maertens J, Giardina P, Kwiatkowski J, Quarta G, Jeng M, Forni GL, Stadler M, Cario H, Debusscher L, Della Porta M, Cazzola M, Greenberg P, Alimena G, Rabault B, Gathmann I, Ford JM, Alberti D, Rose C. Relative response of patients with myelodysplastic syndromes and other transfusion-dependent anaemias to deferasirox (ICL670): a 1-yr prospective study. Eur J Haematol 2007; 80:168-76. PubMed
  • Odegard KC, Zurakowski D, Hornykewycz S, DiNardo JA, Castro RA, Neufeld EJ, Laussen PC. Evaluation of the coagulation system in children with two-ventricle congenital heart disease. Ann Thorac Surg 2007; 83:1797-803. PubMed
  • Klaassen RJ, Blanchette VS, Barnard D, Wakefield CD, Curtis C, Bradley CS, Neufeld EJ, Buchanan GR, Silva MP, Chan AK, Young NL. Validity, reliability, and responsiveness of a new measure of health-related quality of life in children with immune thrombocytopenic purpura: the Kids' ITP Tools. J Pediatr 2007; 150:510-5, 515.e1. PubMed
  • Tubman VN, Levine JE, Campagna DR, Monahan-Earley R, Dvorak AM, Neufeld EJ, Fleming MD. X-linked gray platelet syndrome due to a GATA1 Arg216Gln mutation. Blood 2007; 109:3297-9. PubMed
  • El-Hallak M, Binstadt BA, Leichtner AM, Bennett CM, Neufeld EJ, Fuhlbrigge RC, Zurakowski D, Sundel RP. Clinical effects and safety of rituximab for treatment of refractory pediatric autoimmune diseases. J Pediatr 2007; 150:376-82. PubMed
  • Giulino LB, Bussel JB, Neufeld EJ, . Treatment with rituximab in benign and malignant hematologic disorders in children. J Pediatr 2007; 150:338-44, 344.e1. PubMed
  • Kearney SL, Nemeth E, Neufeld EJ, Thapa D, Ganz T, Weinstein DA, Cunningham MJ. Urinary hepcidin in congenital chronic anemias. Pediatr Blood Cancer 2005; 48:57-63. PubMed
  • Almond CS, Harrington J, Thiagarajan R, Duncan CN, LaPierre R, Halwick D, Blume ED, Del Nido PJ, Neufeld EJ, McGowan FX. Successful use of bivalirudin for cardiac transplantation in a child with heparin-induced thrombocytopenia. J Heart Lung Transplant 2006; 25:1376-9. PubMed
  • Bennett CM, de Jong JL, Neufeld EJ. Targeted ITP strategies: do they elucidate the biology of ITP and related disorders? Pediatr Blood Cancer 2006; 47:706-9. PubMed