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Research Articles

Compiled for your convenience are articles on bone marrow failure research and treatment from the world’s major journals on hematology / oncology.

Article Title Original Publication Date Journal Bone Marrow Disease
HLA-haploidentical stem cell transplantation in children with inherited bone marrow failure syndromes: A retrospective analysis on behalf of EBMT severe aplastic Anemia and pediatric diseases working parties Jun 2024 American Journal of Hematology Aplastic Anemia
Iptacopan and danicopan for paroxysmal nocturnal hemoglobinuria Jun 2024 Journal of Managed Care & Specialty Pharmacy Paroxysmal Nocturnal Hemoglobinuria (PNH)
Complement inhibition in paroxysmal nocturnal hemoglobinuria: From biology to therapy May 2024 International Journal of Laboratory Hematology Paroxysmal Nocturnal Hemoglobinuria (PNH)
Romiplostim for Treatment of Children and Young Adults With Severe Aplastic Anemia and Myelodysplastic Syndrome May 2024 Journal of Pediatric Hematology/Oncology Aplastic Anemia
Toward a more patient-centered drug development process in clinical trials for patients with myelodysplastic syndromes/neoplasms (MDS): Practical considerations from the International Consortium for MDS (icMDS) May 2024 HemaSphere Myelodysplastic Syndromes (MDS)
Exploring treatment strategies for paroxysmal nocturnal hemoglobinuria: an overview of registered clinical trials May 2024 Current Medical Research and Opinion Paroxysmal Nocturnal Hemoglobinuria (PNH)
Treatment of newly diagnosed severe aplastic anemia in children: Evidence-based recommendations May 2024 Pediatric Blood & Cancer Aplastic Anemia
Clinical and Genomic-Based Decision Support System to Define the Optimal Timing of Allogeneic Hematopoietic Stem-Cell Transplantation in Patients With Myelodysplastic Syndromes May 2024 Journal of Clinical Oncology Myelodysplastic Syndromes (MDS)
Advances in the management of higher-risk myelodysplastic syndromes: future prospects May 2024 Leukemia & Lymphoma Myelodysplastic Syndromes (MDS)
Molecular and clinical presentation of UBA1-mutated myelodysplastic syndromes Apr 2024 Blood Myelodysplastic Syndromes (MDS)