A case-based discussion of clinical problems in the management of patients treated with ruxolitinib for myelofibrosis.

P. Joy Ho, Ashish Bajel, Kate Burbury, Lindsay Dunlop, Simon Durrant, Cecily Forsyth, Andrew Perkins, David Ross

Research output: Contribution to journalArticlepeer-review

2 Citations (Scopus)


Ruxolitinib is a dual janus kinase 1 (JAK1)/JAK2 inhibitor used to treat splenomegaly and symptoms associated with myelofibrosis (MF). Current therapeutic options for symptomatic MF include supportive care, myelosuppressive therapy (such as hydroxycarbamide) and janus kinase (JAK) inhibitors (in particular ruxolitinib). Allogeneic stem cell transplantation remains the only potentially curative treatment for MF, and younger transplant-eligible patients should still be considered for allogeneic stem cell transplantation; however, this is applicable only to a small proportion of patients. There is now increasing and extensive experience of the efficacy and safety of ruxolitinib in MF, both in clinical trials and in ‘real-world’ practice. The drug has been shown to be of benefit in intermediate-1 risk patients with symptomatic splenomegaly or other MF-related symptoms, and higher risk disease. Optimal use of the drug is required to maximise clinical benefit, requiring an understanding of the balance between dose-dependent responses and dose-limiting toxicities. There is also increasing experience in the use of ruxolitinib in the pre-transplantation setting. This paper aims to utilise several ‘real-life’ cases to illustrate several strategies that may help to optimise clinical practice.

Original languageEnglish
Pages (from-to)262-268
Number of pages7
JournalInternal Medicine Journal
Issue number3
Publication statusPublished - 1 Mar 2017

Bibliographical note

Funding Information:
We thank Dr Belinda Butcher, CMPP (WriteSource Medical Pty Ltd), who provided medical writing services that were funded by an unrestricted educational grant from Novartis.

Publisher Copyright:
© 2017 Royal Australasian College of Physicians

Copyright 2017 Elsevier B.V., All rights reserved.


  • drug-related side effects and adverse reactions
  • janus kinases
  • primary myelofibrosis
  • prognosis
  • ruxolitinib


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