Allo-HSCT for the treatment of pediatric myelofibrosis

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.

Overall, 48.6% of patients received cells from a matched sibling donor (MSD) and 51.4% received cells from a non-MSD.

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%.

According to univariate analysis, donor type did not influence outcomes including relapse incidence, PFS, GRFS, and OS.

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).

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.