Allogeneic hematopoietic cell transplantation for myelofibrosis: A 10‐year experience at single institution

Abstract

Allogeneic hematopoietic cell transplantation (HCT) remains the only curative approach for patients with myelofibrosis [1,2]; however, it can result in substantial transplant related mortality. We retrospectively examined the outcomes of 18 myelofibrosis patients who underwent allogeneic HCT between 2000 and 2008. Eleven (61%) had high-risk prognostic scores. Fourteen (78%) received a reduced-toxicity regimen of pharmacokinetically-targeted (a median AUC of 5,300 μm min ―1 ; range, 3,500-6,000) intravenous busulfan plus fludarabine (t-IV Bu/Flu). Source of hematopoietic cells were from HLA matched-related (50%), matched-unrelated (44%), or mismatched-unrelated (6%) donors. Fourteen (78%) received G-CSF-mobilized peripheral blood stem cells. Median time to neutrophil and platelet engraftment were 17 (range, 15-28) and 18 (range, 8-213) days, respectively. There were two cases of secondary graft failure after myeloablative conditioning and one case following t-IV Bu/Flu. Overall survival was 50.2% at 54 months (95% confidence interval [C: 24.1-71.6%) with a median follow-up of 15 (range, 3-63) months. Nonrelapsed mortality (NRM) at 100 days and at 1-year were 0 and 22.6% (95% CI: 9.1-49.7%), respectively. The causes of NRM comprised of infections (56%) and graft-versus-host-disease (44%). t-IV Bu/Flu is a feasible conditioning regimen for allogeneic HCT in myelofibrosis showing encouraging engraftment with 7% graft failure rate.