Standard care and investigational drugs in the treatment of myelofibrosis

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Daniela Barraco MD, Margherita Maffioli MD, Francesco Passamonti MD

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Myelofibrosis (MF) is a heterogeneous disorder characterized by splenomegaly, constitutional symptoms, ineffective hematopoiesis, and an increased risk of leukemic transformation. The ongoing research in understanding the pathophysiology of the disease has allowed for the development of targeted drugs optimizing patient management. Furthermore, disease prognostication has significantly improved. Current therapeutic interventions are only partially effective with only allogeneic stem cell transplant potentially curative. Ruxolitinib is the only approved therapy for MF by the US Food and Drug Administration. However, despite efficacy in reducing splenomegaly and controlling symptomatology, it is not associated with consistent molecular or pathologic responses. Drug discontinuation is associated with a dismal outcome. The therapeutic landscape in MF has significantly improved, and emerging drugs with different target pathways, alone or in combination with ruxolitinib, seem promising.

Keywords: anemia, JAK inhibitors, myelofibrosis, survival, treatment.

Citation: Barraco D, Maffioli M, Passamonti F. Standard care and investigational drugs in the treatment of myelofibrosis. Drugs in Context 2019; 8: 212603. DOI: 10.7573/dic.212603

Contributions: DB and MM share first authorship. All authors contributed equally to the preparation of this review. All named authors meet the International Committee of Medical Journal Editors (ICMJE) criteria for authorship for this article, take responsibility for the integrity of the work as a whole, and have given their approval for this version to be published.

Disclosure and potential conflicts of interest: The authors declare that they have no conflicts of interest. The International Committee of Medical Journal Editors (ICMJE) Potential Conflicts of Interests form for the authors is available for download at

Acknowledgments: None.

Funding declaration: We were funded by Fondazione Regionale Ricerca Biomedica, Milan, Italy [FRRB project no. 2015-0042, Genomic profiling of rare hematologic malignancies, development of personalized medicine strategies, and their implementation into the Rete Ematologica Lombarda (REL) clinical network], by the Fondazione Matarelli (Milano, Italy), Fondazione Rusconi (Milano, Italy) and AIL Varese ONLUS.

Copyright: Copyright © 2019 Barraco D, Maffioli M, Passamonti F. Published by Drugs in Context under Creative Commons License Deed CC BY NC ND 4.0 which allows anyone to copy, distribute, and transmit the article provided it is properly attributed in the manner specified below. No commercial use without permission.

Correct attribution: Copyright © 2019 Barraco D, Maffioli M, Passamonti F. Published by Drugs in Context under Creative Commons License Deed CC BY NC ND 4.0.

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Correspondence: Daniela Barraco, MD, Hematology, Department of Medicine and Surgery, University of Insubria, ASST Sette Laghi, Varese, Italy.

Provenance: invited; externally peer reviewed.

Submitted: 7 June 2019; Peer review comments to author: 22 July 2019; Revised manuscript received: 23 August 2019; Accepted: 29 August 2019; Publication date: 26 September 2019.

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