Stem cell transplant for the treatment of myelofibrosis

Introduction: Allogeneic hematopoietic stem cell transplantation (HSCT) remains the cornerstone of curative approach to myelofibrosis (MF), although it is burdened by not negligible toxicity and mortality. Areas covered: In this review, authors discuss the status-of-the-art of HSCT in MF, emphasizing the current limits and the areas for improvement. We interrogated public databases for papers published in the last 30 years. Expert opinion: The therapeutic landscape of MF has been revolutionized after the approval of JAK inhibitor ruxolitinib, which showed impressive efficacy in reducing splenomegaly and ameliorating symptoms and quality of life. Unfortunately, the disease-modifying activity of ruxolitinib is modest, with most patients ultimately dying due to disease progression. Identification of potential candidates to HSCT is critical in order to balance risks and expected benefits, and should rely on risk scores specifically developed to such purpose. The use of ruxolitinib as bridge to HSCT might increase the proportion of patients ultimately able to undergo the procedure and possibly improve their outcome, and represents an important area of research. Since MF is a disease of middle age, further improvements should aim to reduce toxicity of the HSCT procedure and expand the use of alternative, particularly haploidentical, donors.