Symposium | Navigating therapeutic options for patients with cytopenic MF

At the ESH 11th Translational Research Conference: Myeloproliferative Neoplasms, April 24–26, 2026, Estoril, PT, the MPN Hub held a symposium, titled Patient-centered approaches in myelofibrosis: Tailoring treatment for anemia and thrombocytopenia. During the symposium, MPN Hub Steering Committee member Steffen Koschmieder, Uniklinikum RWTH Aachen, Aachen, DE, delivered a presentation on navigating therapeutic options for patients with cytopenic myelofibrosis (MF).

In this presentation, Koschmieder discusses current therapeutic approaches to cytopenic MF, including a review of key data on outcomes with momelotinib, pacritinib, luspatercept, and pelabresib and selinexor in combination with ruxolitinib. 

Key points 

• The management of cytopenic MF requires balancing the treatment of cytopenias with MF-directed therapy. Although cytopenias have traditionally been managed with supportive approaches, targeted therapies for cytopenias and cytopenic MF have recently emerged (Figure 1).^1–5 
• Two targeted therapies are currently approved for the treatment of patients with cytopenic MF. 
• Momelotinib is approved in the US for adult patients with intermediate or high-risk MF, including primary MF, post-polycythemia vera (PV) MF, or post-essential thrombocythemia (ET) MF, with anemia, and in the EU for splenomegaly or other disease-related symptoms in adult patients with moderate to severe anemia who have primary MF, post-PV MF, or post-ET MF and who are Janus kinase inhibitor (JAKi)-naïve or have been treated with ruxolitinib.^6,7 
• Pacritinib is approved in the US for adult patients with intermediate or high-risk MF, including primary MF, post-PV MF, or post-ET MF, with a platelet count <50 × 10⁹/L.⁸ 

• Momelotinib maintained spleen and symptom control while improving transfusion outcomes across the phase III SIMPLIFY-1 (NCT01969838; N = 432), SIMPLIFY-2 (NCT02101268; N = 156), and MOMENTUM (NCT04173494; N = 195) trials.^9–11 
• Across these trials, momelotinib maintained or improved hemoglobin levels over time, and 77–87% of patients maintained or improved transfusion intensity vs baseline.^9-12 
• In a post hoc analysis of the MOMENTUM trial, efficacy was observed across platelet subgroups, including <50 × 10⁹/L (Figure 2), suggesting that the activity of momelotinib may extend beyond anemia to include patients with thrombocytopenia.¹³ 

• Pacritinib improved clinical outcomes in patients with MF and thrombocytopenia in the phase III PERSIST-2 trial (NCT02055781), with additional evidence of anemia benefit demonstrated by a sub-analysis of transfusion independence rates.^14–16 
• Findings from the phase II ACE-536-MF-001 (NCT03194542) and phase II ODYSSEY (NCT06517875) trials demonstrated that luspatercept improved anemia outcomes in patients with MF, with or without JAK inhibitor therapy.^17-19 
• In the phase III MANIFEST-2 trial (NCT04603495), the addition of pelabresib to ruxolitinib was associated with increased thrombocytopenia and reduced anemia compared with ruxolitinib monotherapy.^21,21 
• Initial topline results from the phase III SENTRY trial (NCT04562389) demonstrated that selinexor in combination with ruxolitinib improved spleen responses (47% vs 23% at Week 36), with similar symptom improvement vs ruxolitinib monotherapy but with frequent cytopenias (thrombocytopenia, 59% vs 43%; anemia, 57% vs 58%).²² 
• Emerging therapies may directly improve cytopenias or allow reduced JAK inhibitor dosing without sacrificing efficacy in MF.^2–5 

This educational resource is independently supported by GSK. All content is developed by the faculty in collaboration with SES. Funders are allowed no influence.