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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
Treatment of newly diagnosed severe aplastic anemia in children: Evidence-based recommendations May 2024 Pediatric Blood & Cancer 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)
Romiplostim for Treatment of Children and Young Adults With Severe Aplastic Anemia and Myelodysplastic Syndrome May 2024 Journal of Pediatric Hematology/Oncology Aplastic Anemia
Complement inhibition in paroxysmal nocturnal hemoglobinuria: From biology to therapy May 2024 International Journal of Laboratory Hematology Paroxysmal Nocturnal Hemoglobinuria (PNH)
Haploidentical stem cell transplantation (haplo-SCT) represents the main alternative for children with inherited bone marrow failure syndrome (I-BMF) lacking a matched donor. This retrospective study, conducted on behalf of the EBMT SAAWP and PDWP, aims t Jun 2024 American Society of Clinical Oncology Educational Book Myelodysplastic Syndromes (MDS)
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
Granulocyte transfusions in severe aplastic anemia Jun 2024 Haematologica Aplastic Anemia
Iptacopan and danicopan for paroxysmal nocturnal hemoglobinuria Jun 2024 Journal of Managed Care & Specialty Pharmacy Paroxysmal Nocturnal Hemoglobinuria (PNH)
Oral Iptacopan in Paroxysmal Nocturnal Hemoglobinuria. Reply Jun 2024 The New England Journal of Medicine Paroxysmal Nocturnal Hemoglobinuria (PNH)
Oral Iptacopan in Paroxysmal Nocturnal Hemoglobinuria Jun 2024 The New England Journal of Medicine Paroxysmal Nocturnal Hemoglobinuria (PNH)