Treatment of myelofibrosis: Old and new strategies

Alessandra Iurlo, Daniele Cattaneo

Research output: Contribution to journalArticlepeer-review

Abstract

Myelofibrosis (MF) is a BCR-ABL1–negative myeloproliferative neoplasm that is mainly characterised by reactive bone marrow fibrosis, extramedullary haematopoiesis, anaemia, hepatosplenomegaly, constitutional symptoms, leukaemic progression, and shortened survival. As such, this malignancy is still orphan of curative treatments; indeed, the only treatment that has a clearly demonstrated impact on disease progression is allogeneic haematopoietic stem cell transplantation, but only a minority of patients are eligible for such intensive therapy. However, more recently, the discovery of JAK2 mutations has also led to the development of small-molecule JAK1/2 inhibitors, the first of which, ruxolitinib, has been approved for the treatment of MF in the United States and Europe. In this article, we report on old and new therapeutic strategies that proved effective in early preclinical and clinical trials, and subsequently in the daily clinical practice, for patients with MF, particularly concerning the topics of anaemia, splenomegaly, iron overload, and allogeneic stem cell transplantation.

Original languageEnglish
JournalClinical Medicine Insights: Blood Disorders
Volume10
DOIs
Publication statusPublished - Mar 8 2017

Keywords

  • Allogeneic stem cell transplantation
  • JAK2 inhibitors
  • Momelotinib
  • Myelofibrosis
  • Myeloproliferative neoplasms
  • Ruxolitinib

ASJC Scopus subject areas

  • Hematology

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