Hematopoietic stem cell transplantation offers a curative therapy for patients with myelofibrosis. Because of toxicity, allografting following myeloablative regimens is mainly applicable to young patients. With the introduction of dose-reduced conditioning using busulfan or melphalan with fludarabine, transplantation has become tolerable also for older patients. Implementation of antithymocyte globulin in the conditioning has resulted in effective prevention of graft-versus-host disease and an increased use of alternative donors. Through the discovery of new disease-specific mutations, close monitoring of residual disease became feasible in many patients and the outcome of posttransplant strategies improved. Still challenging is the achievement of new, transplant-derived models to estimate risk status and possible outcome for every individual patient, to help in therapeutic decision making and the determination of the optimal timing of stem cell transplantation. Such a tool may optimally include not only clinicomorphologic characteristics but also other potentially relevant factors such as cytogenetics and novel molecular markers.
