Allogeneic stem-cell transplantation for myelofibrosis

Allogeneic hematopoietic stem-cell transplantation (HSCT) remains the only curative therapy for myelofibrosis. The number of HSCTs performed for this indication has been steadily increasing over the past years, even after the approval of the Janus kinase (JAK) inhibitor, ruxolitinib. This increase may be attributed to improved patient selection based on new prognostic molecular markers, more frequent use of matched unrelated donors, secondary to better (high-resolution) human leukocyte antigen typing and supportive care. Ruxolitinib approval raises new questions regarding the role of JAK inhibitors in the transplant setting.

The current review summarizes recent updates on HSCT in myelofibrosis. Predictors for transplant outcomes, and specific considerations related to myelofibrosis patient selection for HSCT (e.g. molecular risk stratification) are reviewed. In addition, this review will consider management of myelofibrosis patients in the peritransplant period, including the role of ruxolitinib in the pretransplant period, pre and posttransplant splenomegaly, transplant protocols, posttransplant follow-up of minimal residual disease and interventions in the event of poor engraftment.

HSCT remains a highly relevant treatment option for myelofibrosis in the era of JAK inhibitors. Recent advances may contribute to a refined definition of HSCT eligibility and identification of the optimal transplantation time, conditioning protocols and posttransplant management.