Low doses of posttransplant cyclophosphamide reduced the incidence of graft versus host disease in patients with high-risk myelofibrosis (MF) without causing graft failure, according to a new study published in the journal Transplantation and Cellular Therapy.
More research is needed to confirm these results, noted Irene García-Cadenas, MD, PhD, and colleagues.
Read more about treatments for MF
Their aim was to assess the efficacy of reduced doses of posttransplant cyclophosphamide (60 mg/kg total) and tacrolimus as graft versus host disease prophylaxis.
The team analyzed a total of 14 patients with MF who had had hematopoietic-cell transplantation. All patients had been treated with ruxolitinib and the majority (71%) did not respond. Most patients (78%) had splenomegaly at the time of hematopoietic-cell transplantation.
The researchers reported that there were no graft failures. Neutrophil recovery occurred at a median of 21 days and platelet recovery occurred at a median of 31 days.
The cumulative incidence of grade 2 to 4 acute graft versus host disease was 28.6% while the cumulative incidence of grade 3 to 4 acute graft versus host disease was 7%.
The 2-year incidence of overall chronic graft versus host disease was 36% while that of moderate to severe chronic graft versus host disease was 14%.
Only 1 patient relapsed following the transplant. The nonrelapse mortality rate at 100 days was 7% and the nonrelapse mortality rate at 2 years was 14%.
Graft versus host disease-free, immunosuppression-free, and relapse-free incidence at 1 year was 41% while the overall and progression-free survival rates were 86% and 69%, respectively.
Hematopoietic stem cell transplantation is the only curative treatment option for patients with MF. However, the treatment is associated with the risk of post-transplant mortality and is usually only considered for patients who are at high risk or intermediate risk.
García-Cadenas I, Redondo S, Esquirol A, et al. Successful outcome in patients with myelofibrosis undergoing allogeneic donor hematopoietic cell transplantation using reduced-doses of post-transplant cyclophosphamide: challenges and review of the literature. Transplant Cell Ther. Published online April 20, 2023. doi:10.1016/j.jtct.2023.04.008