Myelofibrosis management in routine clinical practice with a focus on patients with cytopenias: recommendations from a global consensus group

Myelofibrosis (MF) is a Philadelphia chromosome (BCR::ABL1)negative myeloproliferative neoplasm, a hallmark of which is progressive deposition of fibrotic tissue in bone marrow 1.Clinical manifestations of MF often include splenomegaly, cytopenias (such as severe anemia), and extramedullary hematopoiesis 1.The Janus kinase inhibitor (JAKi) therapies ruxolitinib (RUX) and fedratinib (FED) have demonstrated significant clinical efficacy in splenic volume reduction and symptom improvement, but they may induce treatment-related anemia and thrombocytopenia 23456789.Other JAKi options include pacritinib (PAC), which received FDA approval in 2022 for patients with MF and severe thrombocytopenia (platelet count <50 × 10 9 /l), and momelotinib (MMB), which received FDA and EMA approval in 2023/2024, respectively, for patients with MF and anemia 101112.Clinical trials with JAKis in MF are summarized in reference 1.National and international guidelines exist for the management of MF; however, a need remains for practical guidance applicable in everyday clinical practice, especially for patients experiencing cytopenias or potential failure of current therapy.The landscape is further complicated by the availability of multiple prognostic tools for MF; as such, clinicians may find disease prognostication challenging and confusing.Additionally, to maximize clinical applicability of trial data, inclusivity of eligibility criteria in the context of the real-world MF patient population should be considered.Recognizing these significant challenges, an international expert consensus group was established to provide best practice recommendations for healthcare professionals, intending to supplement, but not replace, existing guidelines.The consensus program was conducted between March and September 2023, led by a Steering Committee (SC) of nine international hematology experts.The program utilized a modified Delphi methodology; the SC performed several rounds of consensus statement review before submission to the Extended Faculty (EF) of hematologists and patients-nominated by the SC -for a decisive vote.Funding was provided by GlaxoSmithKline (GSK), Brentford, UK.Importantly, GSK had no involvement in formulating questions or recommendations, and no representative participated in the voting process.The SC identified 25 key clinical questions across five consensus themes: