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