Publications by authors named "Ellis Neufeld"

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Hospital-Based Careers in Pediatric Hematology-Oncology: The State of the Field and Future Needs.
Ambika Sohal, Jason L Freedman, Steven Aller, Carmen Cobb, Matt Davis, Ellis J Neufeld

Pediatr Blood Cancer· January 2026

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Eltrombopag for Newly Diagnosed Pediatric Immune Thrombocytopenia Requiring Treatment: The PINES Randomized Clinical Trial.
Kristin A Shimano, Amanda B Grimes, Shipra Kaicker, Sanjay J Shah, Elizabeth Gunn, Ellis J Neufeld

JAMA· November 2025

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Taking Care of the Caregivers: A Quality-of-Life Measure for the Parents of Children With Transfusion-Dependent.
Robert J Klaassen, Rania S Khelifi, Nicholas Barrowman, Manuela Merelles-Pulcini, Elliott P Vichinsky, Ellis J Neufeld

Pediatr Blood Cancer· November 2025

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Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B.
Ulrike M Reiss, Andrew M Davidoff, Edward G D Tuddenham, Pratima Chowdary, Jenny McIntosh, Ellis J Neufeld

N Engl J Med· June 2025

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Genetic variants in canonical Wnt signaling pathway associated with pediatric immune thrombocytopenia.
Taylor Olmsted Kim, Jennifer M Geris, Jonathan M Flanagan, Rachael F Grace, Michele P Lambert, Ellis J Neufeld

Blood Adv· November 2024

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Which pediatric ITP patients will go on to lupus?
Ellis J Neufeld

Blood· April 2024

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Diagnosis and management of pyruvate kinase deficiency: international expert guidelines.
Hanny Al-Samkari, Nadine Shehata, Kelly Lang-Robertson, Paola Bianchi, Andreas Glenthøj, Ellis J Neufeld

Lancet Haematol· March 2024

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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, Ellis J Neufeld

Eur J Haematol· October 2023

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Novel therapeutics and future directions for refractory immune thrombocytopenia.
Hanny Al-Samkari, Ellis J Neufeld

Br J Haematol· October 2023

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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, Ellis J Neufeld

Eur J Haematol· July 2023

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Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: facilitating research through infrastructure, workforce, resources and funding.
Margaret V Ragni, Guy Young, Glaivy Batsuli, Emily Bisson, Shannon L Carpenter, Ellis J Neufeld

Expert Rev Hematol· March 2023

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Gene Therapy and Hemophilia: Where Do We Go from Here?
Nancy S Bolous, Nidhi Bhatt, Nickhill Bhakta, Ellis J Neufeld, Andrew M Davidoff

J Blood Med· October 2022

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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

Br J Haematol· January 2022

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Simoctocog Alfa (Nuwiq) in Previously Untreated Patients with Severe Haemophilia A: Final Results of the NuProtect Study.
Ri J Liesner, Aby Abraham, Carmen Altisent, Mark J Belletrutti, Manuel Carcao, Ellis J Neufeld

Thromb Haemost· November 2021

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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, Ellis J Neufeld

Blood· November 2021

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Pyruvate kinase deficiency in children.
Satheesh Chonat, Stefan W Eber, Susanne Holzhauer, Nina Kollmar, D Holmes Morton, Ellis J Neufeld

Pediatr Blood Cancer· September 2021

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Phase 3 randomised trial of eltrombopag versus standard first-line pharmacological management for newly diagnosed immune thrombocytopaenia (ITP) in children: study protocol.
Kristin A Shimano, Rachael F Grace, Jenny M Despotovic, Ellis J Neufeld, Robert J Klaassen

BMJ Open· August 2021

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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

Haemophilia· May 2021

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Comorbidities and complications in adults with pyruvate kinase deficiency.
Audra N Boscoe, Yan Yan, Elizabeth Hedgeman, Eduard J van Beers, Hanny Al-Samkari, Ellis J Neufeld

Eur J Haematol· April 2021

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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 M Kuo, Ellis J Neufeld

N Engl J Med· March 2020

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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, Ellis J Neufeld

Blood Adv· December 2019

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Second-line treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes.
Rachael F Grace, Kristin A Shimano, Rukhmi Bhat, Cindy Neunert, James B Bussel, Ellis J Neufeld

Am J Hematol· July 2019

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Regional variation and cost implications of prescribed extended half-life factor concentrates among U.S. Haemophilia Treatment Centres for patients with moderate and severe haemophilia.
Stacy E Croteau, Dunlei Cheng, Alice J Cohen, Chris E Holmes, Lynn M Malec, Ellis J Neufeld

Haemophilia· July 2019

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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, Ellis J Neufeld

Haematologica· February 2019

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