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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) |
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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 |
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Iptacopan and danicopan for paroxysmal nocturnal hemoglobinuria |
Jun 2024 |
Journal of Managed Care & Specialty Pharmacy |
Paroxysmal Nocturnal Hemoglobinuria (PNH) |
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Granulocyte transfusions in severe aplastic anemia |
Jun 2024 |
Haematologica |
Aplastic Anemia |
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Complement inhibition in paroxysmal nocturnal hemoglobinuria: From biology to therapy |
May 2024 |
International Journal of Laboratory Hematology |
Paroxysmal Nocturnal Hemoglobinuria (PNH) |
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Romiplostim for Treatment of Children and Young Adults With Severe Aplastic Anemia and Myelodysplastic Syndrome |
May 2024 |
Journal of Pediatric Hematology/Oncology |
Aplastic Anemia |
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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) |
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Treatment of newly diagnosed severe aplastic anemia in children: Evidence-based recommendations |
May 2024 |
Pediatric Blood & Cancer |
Aplastic Anemia |
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Exploring treatment strategies for paroxysmal nocturnal hemoglobinuria: an overview of registered clinical trials |
May 2024 |
Current Medical Research and Opinion |
Paroxysmal Nocturnal Hemoglobinuria (PNH) |
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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) |
