Controversies and dilemmas in allogeneic transplantation for myelofibrosis

Best Pract Res Clin Haematol. 2014 Jun;27(2):165-74. doi: 10.1016/j.beha.2014.07.007. Epub 2014 Jul 19.

Abstract

JAK1/2 inhibitors have broadened the therapeutic options in myelofibrosis. Though not curative, they result in a meaningful clinical benefit with relatively fewer side effects. In contrast, allogeneic hematopoietic cell transplantation (HCT) is a potentially curative option, but is associated with significant morbidity and mortality. Hence, an important question is the optimal timing of HCT in the era of JAK inhibitors. Timing of HCT is a crucial decision, and need to be individualized based on the personal preferences and goals of therapy; in addition to patient, disease, and transplant related factors. Risk stratification by the currently established prognostic scoring systems need to be further refined by incorporation of prognostically significant mutations to guide the treatment choices better. Data on use of JAK inhibitors prior to HCT have just started to emerge. We discuss some of the current controversies and dilemmas in transplantation for myelofibrosis based on a few real life scenarios.

Keywords: CALR mutation; JAK inhibitors; allogeneic transplantation; leukemic transformation; myelofibrosis; prognosis.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Aged
  • Antineoplastic Agents / therapeutic use*
  • Cell Transformation, Neoplastic / drug effects
  • Cell Transformation, Neoplastic / genetics
  • Cell Transformation, Neoplastic / pathology
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Janus Kinase 2 / genetics
  • Middle Aged
  • Mutation
  • Precision Medicine
  • Primary Myelofibrosis / diagnosis
  • Primary Myelofibrosis / genetics
  • Primary Myelofibrosis / pathology
  • Primary Myelofibrosis / therapy*
  • Prognosis
  • Protein Kinase Inhibitors / therapeutic use*
  • Time Factors
  • Transplantation, Homologous
  • Treatment Outcome

Substances

  • Antineoplastic Agents
  • Protein Kinase Inhibitors
  • JAK2 protein, human
  • Janus Kinase 2