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Management of Myelofibrosis-Related Cytopenias

  • Myeloproliferative Neoplasms (B Stein, Section Editor)
  • Published:
Current Hematologic Malignancy Reports Aims and scope Submit manuscript

Abstract

Purpose of Review

Cytopenias, particularly anemia, are frequently encountered in patients with myelofibrosis. Management of cytopenias in myelofibrosis can be very challenging because current therapeutic interventions are only of modest efficacy and ruxolitinib, the only approved drug for myelofibrosis, is myelosuppressive. Yet, dose optimization of ruxolitinib is important for its survival benefit in patients with advanced disease. We sought to summarize the data on treatments for cytopenias available at present and review promising agents in development and emerging strategies.

Recent Findings

The activin receptor ligand traps hold considerable promise for the treatment of anemia and could represent an attractive combination strategy with ruxolitinib. Low-dose thalidomide, which could offset both anemia and thrombocytopenia caused by ruxolitinib, represents another potential partner for ruxolitinib. The anti-fibrotic agent PRM-151 produced sustained improvements in cytopenias in some patients, and further data on this drug are eagerly awaited. Finally, several preclinical leads with translational potential are worthy of clinical investigation as strategies to halt/reverse bone marrow fibrosis and thereby improve cytopenias.

Summary

Cytopenias remain a significant hurdle in myelofibrosis management, but several novel investigational agents hold considerable promise for the future.

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Authors and Affiliations

  1. Department of Leukemia, University of Texas MD Anderson Cancer Center, 1400 Holcombe Blvd, FC4.3062, Unit 428, Houston, TX, 77030, USA

    Prithviraj Bose & Srdan Verstovsek

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  1. Prithviraj Bose
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  2. Srdan Verstovsek
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Correspondence to Prithviraj Bose.

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Conflict of Interest

Prithviraj Bose reports grants and personal fees from Incyte Corporation, grants from CTI BioPharma, and grants from Celgene Corporation, outside the submitted work. Srdan Verstovsek declares that he has no conflict of interest.

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This article does not contain any studies with human or animal subjects performed by any of the authors.

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This article is part of the Topical Collection on Myeloproliferative Neoplasms

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Bose, P., Verstovsek, S. Management of Myelofibrosis-Related Cytopenias. Curr Hematol Malig Rep 13, 164–172 (2018). https://doi.org/10.1007/s11899-018-0447-9

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