Transplant in patients MF: the next best approach after matched donor allo-HSCT

Allogeneic hematopoietic stem cell transplant (allo-HSCT) is currently the only curative treatment option for patients with myelofibrosis (MF). In the absence of a human leukocyte antigen (HLA)-matched donor, cord blood transplant (CBT) or HLA-haploidentical transplant (haplo-HSCT) provide other options for these patients – although the best of these options remains unclear.¹

Here, we summarize a retrospective study by Sakatoku et al.¹ published in Nature on February 20, 2024, comparing CBT and haplo-HSCT as alternative transplant options for MF.

Study design¹

• This multicenter study analyzed patient data from the Japanese Society for Transplantation and Cellular Therapy between 2003 and 2019.
• Patients were undergoing HSCT for the first time to treat MF.

Key findings¹

• Overall, 77 patients with MF were included, with 27 undergoing haplo-HSCT and 50 undergoing CBT.
• The median age was 59 years, and conditioning intensity before transplant was similar in both groups.
• More patients were male than female in both groups (78% and 70%, respectively).
• Most patients had not been treated by a Janus kinase inhibitor prior to transplant in both groups (59% and 74%, respectively).

Results¹

• The median time to neutrophil recovery was longer in CBT recipients compared with haplo-HSCT recipients (27 vs 17 days).
  
• However, final neutrophil engraftment and survival responses were comparable between groups (Figure 1).

CBT, cord blood transplant; CI, cumulative incidence, GvHD, graft-versus-host disease; HSCT, hematopoietic stem cell transplant; MF, myelofibrosis; NRM, non-relapse mortality; OS; overall survival. 
*Adapted from Sakatoku K, et al.¹. 
†Defined as the patient being alive at 28 days with an absolute neutrophil count <0.5 × 109/L. 
‡After 3 years. 

 

• Other studies report similar findings, as captured in a recent report from the European Society for Blood and Marrow Transplantation.

Key learnings
In line with previous studies’ findings in both MF and acute leukemia, these data continue to support haplo-HSCT and CBT as options for allo-HSCT in patients with MF without an HLA-matched donor. Further investigations with larger sample sizes are needed to further elucidate the differences in outcomes between these options.