Experts Reach Consensus on Myelofibrosis Management, Offering New Hope for Patients

A comprehensive international consensus program has provided vital clinical recommendations to enhance the treatment landscape for myelofibrosis (MF), according to an article published in Leukemia.

This rare, Philadelphia chromosome (BCR::ABL1)-negative myeloproliferative neoplasm is marked by bone marrow fibrosis, leading to complex clinical challenges such as splenomegaly, severe anemia, and thrombocytopenia. Despite advancements, significant gaps in guidance persist, particularly for patients with cytopenias or those not responding adequately to current therapies.

“The landscape is further complicated by the availability of multiple prognostic tools for MF; as such, clinicians may find disease prognostication challenging and confusing,” explained Steffen Koschmieder, Department of Hematology, Oncology, Hemostaseology, and Stem Cell Transplantation, Faculty of Medicine, RWTH Aachen University, and Center for Integrated Oncology Aachen Bonn Cologne Düsseldorf (CIO ABCD) in Aachen, Germany, and coauthors.

To address these gaps, an international panel of hematology experts convened between March and September 2023. The program utilized a modified Delphi method, with 9 experts forming the Steering Committee (SC) and collaborating with 20 hematologists and 9 patients in the Extended Faculty (EF). The panel focused on 5 themes: thresholds for anemia and thrombocytopenia treatment, defining JAK inhibitor (JAKi) therapy failure, determining patient prognosis, and identifying unmet needs in MF clinical trials.

The rigorous process involved systematic literature reviews and iterative voting rounds. Consensus required ≥75% agreement on a 9-point scale, achieved for all 15 initial clinical recommendations. The SC amended 9 of these recommendations based on feedback, leading to final approval across the panel. The outcomes emphasize strategies for managing anemia and thrombocytopenia, with specific attention to recently approved JAKi treatments such as pacritinib and momelotinib.

Key findings include the importance of comprehensive anemia workups and considering combination therapies, such as those involving ruxolitinib (RUX), to manage treatment-related anemia. Pacritinib (approved in 2022) and momelotinib (approved in 2023/2024) were highlighted for their roles in severe thrombocytopenia and anemia, respectively.

Patient perspectives played a crucial role, underscoring the significance of communication, trust, and shared decision-making. This inclusive approach ensured that the recommendations accounted for quality of life concerns and patient engagement in their treatment.

While the consensus program faced challenges, such as reduced participation in later voting rounds, it succeeded in delivering robust, patient-informed clinical guidance. These recommendations provide a practical, supplementary framework to existing guidelines, equipping clinicians with enhanced tools for improving MF patient care and outcomes.

“The recommendations presented herein provide an up-to-date overview of contemporary MF treatment, offering a valuable supplement to existing treatment guidelines without aiming to replace them,” wrote the study authors.

Reference

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