Allogeneic hematopoietic cell transplantation for patients with myelofibrosis

Hematopoietic cell transplantation (HCT) offers potentially curative therapy for patients with myelofibrosis. What is the current status?

Changes in transplant strategies allow offering HCT to patients who, because of age or comorbid conditions, were not considered transplant candidates in the past. The omission of high-dose total body irradiation, adjusting doses of busulfan to achieve defined target levels, using fludarabine instead of cyclophosphamide as an immunosuppressive agent, the addition of melphalan, and the incorporation of antithymocyte globulin all appear to have contributed to better tolerability of new regimens. Reduced-intensity conditioning regimens are associated with a decrease in nonrelapse mortality and allow for successful HCT, even in patients 60-70 years of age. Some 50-75% of patients are cured by HCT. Emerging concepts include new prognostic scoring systems and novel molecular markers such as Janus kinase 2 mutations, which may aid in making treatment decisions and assessing remission status.

Modifications of transplant-conditioning regimens have reduced transplant-related mortality and allow carrying out successful HCT in increasingly older patients. The selection of patients who should be transplanted, the optimal timing for transplantation, and pretransplant and posttransplant strategies remain challenging problems.