Allogeneic Stem Cell Transplantation in Myelofibrosis

Biol Blood Marrow Transplant. 2017 Sep;23(9):1429-1436. doi: 10.1016/j.bbmt.2017.05.007. Epub 2017 May 10.

Abstract

Myeloproliferative neoplasm (MPN) is a category in the World Health Organization classification of myeloid tumors. BCR-ABL1-negative MPN is a subcategory that includes primary myelofibrosis (MF), post-essential thrombocythemia MF, and post-polycythemia vera MF. These disorders are characterized by stem cell-derived clonal myeloproliferation. Clinically, these diseases present with anemia and splenomegaly and significant constitutional symptoms such as severe fatigue, symptoms associated with an enlarged spleen and liver, pruritus, fevers, night sweats, and bone pain. Multiple treatment options may provide symptom relief and improved survival; however, allogeneic stem cell transplantation (HCT) remains the only potentially curative option. The decision for a transplant is based on patient prognosis, age, comorbidities, and functional status. This review describes the recent data on various peritransplantation factors and their effect on outcomes of patients with MF and new therapeutic areas, such as the use and timing of Janus kinase inhibitors with HCT and gives overall conclusions from the available data in the published literature.

Keywords: Allogeneic stem cell transplantation; Myelofibrosis.

Publication types

  • Review

MeSH terms

  • Cell Proliferation
  • Disease Management
  • Hematopoietic Stem Cell Transplantation*
  • Hematopoietic Stem Cells / immunology
  • Hematopoietic Stem Cells / pathology
  • Histocompatibility Testing
  • Humans
  • Janus Kinase Inhibitors / therapeutic use
  • Polycythemia Vera / immunology
  • Polycythemia Vera / mortality
  • Polycythemia Vera / pathology
  • Polycythemia Vera / therapy*
  • Primary Myelofibrosis / immunology
  • Primary Myelofibrosis / mortality
  • Primary Myelofibrosis / pathology
  • Primary Myelofibrosis / therapy*
  • Splenomegaly / immunology
  • Splenomegaly / mortality
  • Splenomegaly / pathology
  • Splenomegaly / therapy*
  • Survival Analysis
  • Thrombocythemia, Essential / immunology
  • Thrombocythemia, Essential / mortality
  • Thrombocythemia, Essential / pathology
  • Thrombocythemia, Essential / therapy*
  • Tissue Donors / supply & distribution
  • Transplantation, Homologous
  • Treatment Outcome

Substances

  • Janus Kinase Inhibitors