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2024-10-18T12:20:13.000Z

Recommendations for managing MF with a focus on patients with cytopenias from the global consensus group

Oct 18, 2024
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Learning objective: After reading this article, learners will be able to cite a new clinical development in MPN.

An international expert group was established to address the lack of applicable guidance in routine clinical practice for the management of MF.1 The global consensus group published recommendations in Leukemia for the management of MF, with a focus on patients with cytopenias. The recommendations were based on five consensus themes1:

  • Establishing the thresholds for anemia and when to initiate/modify treatment
  • Establishing the threshold for thrombocytopenia and when to initiate/modify treatment
  • Understanding JAKi failure and what would warrant switching treatment
  • Identifying when and how to determine prognosis in patients with MF
  • Highlighting the unmet needs in MF clinical trials 

A steering committee of nine international hematology experts utilized the modified Delphi process with several rounds of consensus review between March 2023 and September 2023.1 Following this, the consensus statements were submitted to the extended faculty of hematologists and patients.1

Key learnings

A comprehensive assessment, including accurate diagnosis and appropriate treatment, is crucial in optimizing the management of MF-related anemia. All available and emerging therapeutic options should be considered for MF- and treatment-related anemia; only momelotinib is specifically approved for MF-related anemia.  

The recommendations from the German Society of Hematology and Medical Oncology and the Society of Thrombosis and Hemostasis Research can serve as a basis for treatment decisions for MF and thrombocytopenia.  

Momelotinib and pacritinib are the only JAKis approved for use in patients with MF and severe thrombocytopenia (platelet count, ≥25 x 109/L and <50 x 109/L, respectively). In addition to JAKi monotherapy, RUX-based combination therapies are effective in managing MF-related anemia. 

Physicians should not be restricted by definitions of R/R and suboptimal response but instead use them to inform the timing of potential treatment switches. The expert group recommended that collaborative efforts should focus on refinement of the RUX failure criteria to distinguish resistance from intolerance, and these definitions should be extended to other JAKis. 

Validated prognostic tools and clinical variables should be used to predict survival benefits and to guide clinical decisions in the management of MF. 

The panel suggested advocating better trial endpoints to increase their relevance and applicability in real-world clinical settings, while highlighting the need for broader inclusion criteria in clinical trials.  

The global consensus group recommendations provide up-to-date practical guidance to supplement the existing MF treatment guidelines. However, further research is needed for comprehensive clinical decision-making.   

Abbreviations: ACVR1, activin A receptor type 1; IACH, International Academy for Clinical Hematology; JAKi, Janus kinase inhibitor; MF, myelofibrosis; MPN, myeloproliferative neoplasms; OS, overall survival; STAT, signal transducer and activator of transcription.

  1. Koschmieder S, Bose P, Ellis MH, et al. Myelofibrosis management in routine clinical practice with a focus on patients with cytopenias: Recommendations from a global consensus group. Leukemia. 2024;38(8):1831-1838. DOI: 10.1038/s41375-024-02330-7 

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