Outcome of allogeneic stem cell transplantation in patients with myelofibrosis

Myelofibrosis, either de novo or following pre-existing hematologic diseases, can be cured by allogeneic hematopoietic stem cell transplantation (SCT), but SCT is associated with significant morbidity and mortality, making the choice and timing of transplantation difficult. In all, 20 patients (seven female and 13 male), with a median age of 45 years (range 22-57 years), with idiopathic myelofibrosis (n = 12), post-thrombocythemic (n = 3) or post-polycythemic (n = 2) myeloid metaplasia or leukemic transformation (n = 3), underwent allogeneic SCT at our center between 1994 and 2003. With regard to the pre-transplant presence of risk factors such as hemoglobin levels < or =10 mg/dl, grade III marrow fibrosis or peripheral blast counts >1%, patients were divided into high- and low-risk groups. The estimated 3-year survival post transplant was 38.5% for all patients. The 3-year probability of survival within the high-risk group (n = 11) characterized by the presence of at least two risk factors was 16%. Low-risk patients (n = 9) with at most one risk factor had an estimated 3-year survival of 67%. Thus, previously defined risk determinants for the outcome of allogeneic transplantation for myelofibrosis may provide useful information facilitating treatment strategies. Our data suggest that transplantation should be taken into consideration before poor prognostic variables develop.