EllisNeufeldMDPhD, FAAP

Hematology • Memphis, TN

Clotting Disorder, Hemophilia & Bleeding Disorder, Sickle Cell & Other Anemias

Clinical Director, EVP and Physician-in-Chief, St. Jude Children's Research Hospital

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Office
St. Jude Children's Research Hospital
262 Danny Thomas Place
Memphis, TN 38105
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Clinical Expertise

Thalassemias, Immune thrombocytopenic purpura, Deep vein thrombosis, Hemophilia, Thrombotic disorders, Hospital administration, Genetic blood disorders, Iron overload

Education & Training

Children's Hospital/Boston Medical CenterFellowship, Pediatric Hematology/Oncology, 1988 - 1990
Children's Hospital/Harvard Medical SchoolFellowship, Medical Genetics and Genomics, 1988 - 1988
Children's Hospital/Boston Medical CenterResidency, Pediatrics, 1986 - 1988
Children's Hospital/Boston Medical CenterInternship, Pediatrics, 1985 - 1986
Washington University in St. Louis School of MedicineClass of 1985

Certifications & Licensure

TN State Medical License 2017 - 2026
MA State Medical License 1987 - 2025
FL State Medical License 2015 - 2017
American Board of Pediatrics Pediatrics

Awards, Honors, & Recognition

William Hathaway Visiting Professor Denver Children's Hospital, 2012
Abelson Visiting Professor Washington University School of Medicine, 2012
Coopersmith Visiting Professor John's Hopkins University, 2012
National President's Award Cooley's Anemia Foundation, 2011
Elected Member The American Society for Clinical Investigation, 2003
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Clinical Trials

Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN) Start of enrollment: 2000 Jul 01
Completed
Combination Therapy Compared With Single-Drug Therapy in Patients With Cardiac Diseases Start of enrollment: 2005 Jun 01
Terminated
Phase 2
Optimal Platelet Dose Strategy for Management of Thrombocytopenia Start of enrollment: 2004 Jul 01
Completed
Phase 3
Rituximab to Treat Severe Hemophilia A Start of enrollment: 2006 Jun 01
Completed
Phase 2
Evaluating the Presence of Blood Clots in People With Heparin-Induced Thrombocytopenia (HIT) (The HOT Study) Start of enrollment: 2008 Jan 01
Terminated
Safety and Effectiveness of Granulocyte Transfusions in Resolving Infection in People With Neutropenia (The RING Study) Start of enrollment: 2008 Apr 01
Completed
Phase 3
Evaluating People With Thalassemia: The Thalassemia Longitudinal Cohort (TLC) Study Start of enrollment: 2007 May 01
Completed
Evaluating the Effectiveness of Adding Rituximab to Standard Treatment for Thrombotic Thrombocytopenic Purpura (TTP) Start of enrollment: 2009 Apr 01
Terminated
Phase 3
Sildenafil to Improve Exercise Capacity in People With Thalassemia and Pulmonary Hypertension Start of enrollment: 2009 Mar 01
Completed
Phase 2, Phase 3
Assessment of Pain in People With Thalassemia Start of enrollment: 2009 Mar 01
Completed
Initial Treatment of Patients With Immune Thrombocytopenic Purpura Start of enrollment: 2010 Jan 01
Terminated
Phase 3
Efficacy of Vorinostat to Induce Fetal Hemoglobin in Sickle Cell Disease Start of enrollment: 2009 Oct 01
Terminated
Phase 2
Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload Start of enrollment: 2009 Nov 01
Completed
Phase 1
Heparin-Induced Thrombocytopenia - Retrospective Analysis of Data on Incidence and Outcomes Study Start of enrollment: 2010 Jun 01
Completed
Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload Start of enrollment: 2010 Aug 13
Completed
Phase 2
Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia Start of enrollment: 2013 Jun 01
Completed
Phase 1, Phase 2
A Phase II Trial of Regadenoson in Sickle Cell Anemia Start of enrollment: 2013 Jul 01
Completed
Phase 2
ATHN 2: Factor Switching Study Start of enrollment: 2015 Sep 01
Completed
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Publications & Presentations

PubMed

Diagnosis and management of pyruvate kinase deficiency: international expert guidelines.
Al-Samkari, H., Shehata, N., Lang-Robertson, K., Bianchi, P., Glenthøj, A., Sheth, S., Neufeld, E., Rees, D., Chonat, S., Kuo, K., Rothman, J., Barcellini, W., van Bee...> ;The Lancet. Haematology. 2024 Mar 1
3 citations
Novel therapeutics and future directions for refractory immune thrombocytopenia.
Hanny Al-Samkari, Ellis J Neufeld> ;British Journal of Haematology. 2023 Oct 1
Simoctocog alfa (Nuwiq®) in previously untreated patients with severe haemophilia A-Final efficacy and safety results from the NuProtect study.
Mary Mathias, Aby Abraham, Mark J Belletrutti, Manuel Carcao, Manuela Carvalho, Hervé Chambost, Anthony K C Chan, Leonid Dubey, Jonathan Ducore, Michael Gattens, Paolo...> ;European Journal of Haematology. 2023 Oct 1
3 citations
Health-related quality of life in patients with β-thalassemia: Data from the phase 3 BELIEVE trial of luspatercept.
Maria Domenica Cappellini, Ali T Taher, Antonio Piga, Farrukh Shah, Ersi Voskaridou, Vip Viprakasit, John B Porter, Olivier Hermine, Ellis J Neufeld, Alexis A Thompson...> ;European Journal of Haematology. 2023 Jul 1
8 citations
Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: facilitating research through infrastructure, workforce...
Margaret V Ragni, Guy Young, Glaivy Batsuli, Emily Bisson, Shannon L Carpenter, Stacy E Croteau, Adam Cuker, Randall G Curtis, Michael Denne, Bruce Ewenstein, Amber Fe...> ;Expert Review of Hematology. 2023 Mar 1
3 citations
Gene Therapy and Hemophilia: Where Do We Go from Here?
Nancy S Bolous, Nidhi Bhatt, Nickhill Bhakta, Ellis J Neufeld, Andrew M Davidoff, Ulrike M Reiss> ;Journal of Blood Medicine. 2022 Jan 1
4 citations
An update on the US adult thalassaemia population: a report from the CDC thalassaemia treatment centres.
John Chapin, Alan R. Cohen, Ellis J. Neufeld, Elliott Vichinsky, Patricia J. Giardina, Jeanne Boudreaux, Binh C. Le, Kristy Kenney, Sean Trimble, Alexis A. Thompson> ;British Journal of Haematology. 2021 Nov 14
2 citations
Phase 3 randomised trial of eltrombopag versus standard first-line pharmacological management for newly diagnosed immune thrombocytopaenia (ITP) in children: study pro...
Kristin A. Shimano, Rachael F. Grace, Jenny M. Despotovic, Ellis J. Neufeld, Robert J. Klaassen, Carolyn M. Bennett, Clement Ma, Wendy B. London, Cindy Neunert> ;BMJ Open. 2021 Aug 1
9 citations
Pyruvate kinase deficiency in children.
Satheesh Chonat, Stefan W. Eber, Susanne Holzhauer, Nina Kollmar, D. Holmes Morton, Bertil Glader, Ellis J. Neufeld, Hassan M. Yaish, Jennifer A. Rothman, Mukta Sharma...> ;Pediatric Blood & Cancer. 2021 Jun 14
65 citations
Asymptomatic and Symptomatic SARS-CoV-2 Infections After BNT162b2 Vaccination in a Routinely Screened Workforce.
Li Tang, Diego R. Hijano, Aditya H. Gaur, Terrence L. Geiger, Ellis J. Neufeld, James M. Hoffman, Randall T. Hayden> ;JAMA. 2021 Jun 22
16 citations
The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study from the United States perspective
Nancy S. Bolous, Yichen Chen, Huiqi Wang, Andrew M. Davidoff, Meenakshi Devidas, Timothy W. Jacobs, Michael M Meagher, Amit C. Nathwani, Ellis J. Neufeld, Bryan A. Pir...> ;Blood. 2021 Nov 4
3 citations
Limited sampling strategies for accurate determination of extended half-life factor VIII pharmacokinetics in severe haemophilia A patients.
Alanna McEneny-King, Pierre Chelle, Margaret H Goggans, Patricia J. Barker, Timothy W. Jacobs, Ellis J. Neufeld, Ulrike M. Reiss, John C. Panetta> ;Haemophilia. 2021 Mar 20
14 citations
Simoctocog Alfa (Nuwiq) in Previously Untreated Patients with Severe Haemophilia A: Final Results of the NuProtect Study
Ri Liesner, Aby Abraham, Carmen Altisent, Mark Belletrutti, Manuel Carcao, Manuela Carvalho, Hervé Chambost, Anthony K.C. Chan, Leonid Dubey, Jonathan M. Ducore, Micha...> ;Thrombosis and Haemostasis. 2021 Feb 13
16 citations
Comorbidities and complications in adults with pyruvate kinase deficiency.
Audra N. Boscoe, Yan Yan, Elizabeth Hedgeman, Eduard J. van Beers, Hanny Al-Samkari, Wilma Barcellini, Stefan W. Eber, Bertil Glader, Hassan M. Yaish, Satheesh Chonat,...> ;European Journal of Haematology. 2021 Jan 24
90 citations
Mobility, clustering, and transport of nerve growth factor in embryonal sensory cells and in a sympathetic neuronal cell line.
Andrea Levi, Yoram Shechter, Ellis J. Neufeld, Joseph Schlessinger> ;Proceedings of the National Academy of Sciences of the United States of America. 1980 Jun 1
13 citations
Bioengineering hemophilia A–specific microvascular grafts for delivery of full-length factor VIII into the bloodstream
Joseph Neumeyer, Ruei-Zeng Lin, Kai Wang, Xuechong Hong, Tien Hua, Stacy E. Croteau, Ellis J. Neufeld, Juan M. Melero-Martin> ;Blood Advances. 2019 Dec 23
111 citations
A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia
M. Domenica Cappellini, Vip Viprakasit, Ali T Taher, Pencho Georgiev, Kevin H. Kuo, Thomas D. Coates, Ersi Voskaridou, Hong-Keng Liew, Idit Pazgal-Kobrowski, Gian Luca...> ;The New England Journal of Medicine. 2020 Mar 25
28 citations
Second-line treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes
, , , , , , , , , , , , , , , , , , , , , , > ;American Journal of Hematology. 2019 Jul 1
31 citations
Regional variation and cost implications of prescribed extended half-life factor concentrates among U.S. Haemophilia Treatment Centres for patients with moderate and s...
Stacy E. Croteau, Dunlei Cheng, Alice J. Cohen, Chris E. Holmes, Lynn M. Malec, Michael Silvey, Courtney D. Thornburg, Allison P. Wheeler, Peter A. Kouides, Leslie Raf...> ;Haemophilia. 2019 Jul 1
Using pharmacokinetics for tailoring prophylaxis in people with hemophilia switching between clotting factor products: A scoping review.
Yu JK, Iorio A, Edginton AN, WAPPS co‐investigators> ;Research and practice in thrombosis and haemostasis. 2019-05-20
32 citations
Prevalence and management of iron overload in pyruvate kinase deficiency: report from the Pyruvate Kinase Deficiency Natural History Study
Eduard J. van Beers, Stephanie van Straaten, D. Holmes Morton, Wilma Barcellini, Stefan W. Eber, Bertil Glader, Hassan M. Yaish, Satheesh Chonat, Janet L. Kwiatkowski,...> ;Haematologica. 2019 Feb 1
22 citations
Physician decision making in selection of second-line treatments in immune thrombocytopenia in children
Rachael F. Grace, Jenny M. Despotovic, Carolyn M. Bennett, James B. Bussel, Michelle Neier, Cindy E. Neunert, Shelley E. Crary, Yves D. Pastore, Robert J. Klaassen, Je...> ;American Journal of Hematology. 2018 Jul 1
5 citations
Recombinant activated factor VII in approved indications: Update on safety.
Ellis J. Neufeld, Claude Negrier, S. Benchikh el Fegoun, David L. Cooper, A. Rojas-Rios, Stephanie Seremetis> ;Haemophilia. 2018 Jul 1
45 citations
Performing and interpreting individual pharmacokinetic profiles in patients with Hemophilia A or B: Rationale and general considerations.
Alfonso Iorio, Andrea N. Edginton, Victor S. Blanchette, Jan Blatny, Ana Boban, Marjon H. Cnossen, Peter William Collins, Stacy E. Croteau, Katheljin Fischer, Daniel P...> ;Research and Practice in Thrombosis and Haemostasis. 2018 Jul 1
11 citations
Focusing in on use of pharmacokinetic profiles in routine hemophilia care.
Stacy E. Croteau, Michael U. Callaghan, Joanna A. Davis, Amy L. Dunn, Michael F. Guerrera, Osman Khan, Ellis J. Neufeld, Leslie Raffini, Michael Recht, Michael Wang, A...> ;Research and Practice in Thrombosis and Haemostasis. 2018 May 27
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Abstracts/Posters

Inhibitor Development with Simoctocog Alfa in Previously Untreated Patients with Severe Haemophilia a: Final Results of the Nuprotect Study
Ellis J. Neufeld, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/9/2019
Comorbidities and Complications in Adults with Pyruvate Kinase Deficiency
Ellis J. Neufeld, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/7/2019
A Phase 3 Study of Eltrombopag Vs. Standard First-Line Management for Newly Diagnosed Immune Thrombocytopenia in Children
Ellis J. Neufeld, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/8/2019
Lack of Inhibitor Development in the American Thrombosis and Hemostasis Network (ATHN)-2 Factor Switching Study: Preliminary Report of Primary Outcome
Ellis J. Neufeld, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/7/2019
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Lectures

Navigating Immune Thrombocytopenic Purpura Treatment in 2018: Expert Perspectives on the Latest Advances
60th American Society of Hematology Annual Meeting - 11/30/2018
2011 ASH Annual Meeting and Exposition
American Society of Hematology (ASH), San Diego, California - 12/10/2011

Press Mentions

U.S. News Ranks Two Memphis Children's Hospitals Among Nation's BestJune 14th, 2022
COVID-19 Vaccine Is Associated with Fewer Asymptomatic SARS-CoV-2 Infections, Study FindsMay 6th, 2021
Vioxx, Once Vilified like Thalidomide, May Also Return to the MarketOctober 10th, 2019
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Hospital Affiliations

St. Jude Children's Research HospitalMemphis, Tennessee
Le Bonheur Children's HospitalMemphis, Tennessee