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Allo-HSCT for the treatment of pediatric myelofibrosis
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A retrospective multicenter study that evaluated the outcomes of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in pediatric patients for the treatment of myelofibrosis (MF) between 2000 and 2022 has been published in Bone Marrow Transplantation by Wachowiak et al.1 Data from the European Society for Blood and Marrow Transplantation (EBMT) registry for 35 children were analyzed, representing the largest pediatric cohort with MF and in receipt of allo-HSCT studied to date. |
| Key learnings1 |
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Overall, 48.6% of patients received cells from a matched sibling donor (MSD) and 51.4% received cells from a non-MSD. |
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In the total cohort, the 6-year overall survival (OS) was 71.1%, progression-free survival (PFS) was 66.1%, non-relapse mortality (NRM) was 18%, and graft-versus-host disease (GvHD)-free relapse-free survival (GFRS) was 50%. |
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According to univariate analysis, donor type did not influence outcomes including relapse incidence, PFS, GRFS, and OS. |
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Bone marrow transplantation resulted in significantly higher 6-year OS and PFS rates than peripheral blood or cord blood transplant (90.9% vs 54%, p = 0.01; and 85.1% vs 50.8%, p = 0.03, respectively), and lower NRM rates (0% vs 32% for transplants with cells from other sources). |
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These data highlight the potential benefits of allo-HCT in pediatric MF and suggest that bone marrow should be the recommended source of hematopoietic cells for allo-HSCT in this population. |
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