Allogeneic haematopoietic cell transplantation for myelofibrosis: a real-life perspective

Br J Haematol. 2021 Nov;195(4):495-506. doi: 10.1111/bjh.17469. Epub 2021 Apr 21.

Abstract

Myelofibrosis (MF) is a clonal stem cell neoplasm with heterogeneous clinical phenotypes and well-established molecular drivers. Allogeneic haematopoietic stem cell transplantation (HSCT) offers an important curative treatment option for primary MF and post-essential thrombocythaemia/polycythaemia vera MF or secondary MF. With a disease course that varies from indolent to highly progressive, we are now able to stratify risk of mortality through various tools including patient-related clinical characteristics as well as molecular genetic profile. Owing to the high risk of mortality and morbidity associated with HSCT for patients with myelofibrosis, it is important to improve patient selection for transplant. Our primary goal is to comprehensively define our understanding of current practices including the role of Janus Kinase (JAK) inhibitors, to present the data behind transplantation before and after leukaemic transformation, and to introduce novel personalization of MF treatment with a proposed clinical-molecular prognostic model to help elucidate a timepoint optimal for consideration of HSCT.

Keywords: allogeneic transplant; myelofibrosis; ruxolitinib; splenectomy.

Publication types

  • Review

MeSH terms

  • Allografts
  • Clinical Trials as Topic
  • Combined Modality Therapy
  • Cyclophosphamide / administration & dosage
  • Cyclophosphamide / therapeutic use
  • Disease Progression
  • Donor Selection
  • Hematopoiesis, Extramedullary
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Janus Kinase 2 / antagonists & inhibitors
  • Janus Kinase 2 / genetics
  • Middle Aged
  • Mutation
  • Nitriles / administration & dosage
  • Nitriles / therapeutic use
  • Premedication
  • Primary Myelofibrosis / drug therapy
  • Primary Myelofibrosis / genetics
  • Primary Myelofibrosis / surgery
  • Primary Myelofibrosis / therapy*
  • Prognosis
  • Protein Kinase Inhibitors / therapeutic use
  • Pyrazoles / administration & dosage
  • Pyrazoles / therapeutic use
  • Pyrimidines / administration & dosage
  • Pyrimidines / therapeutic use
  • Recurrence
  • Risk Assessment
  • Salvage Therapy
  • Severity of Illness Index
  • Splenectomy
  • Transplantation Conditioning / methods

Substances

  • Nitriles
  • Protein Kinase Inhibitors
  • Pyrazoles
  • Pyrimidines
  • ruxolitinib
  • Cyclophosphamide
  • JAK2 protein, human
  • Janus Kinase 2