BackgroundMyelofbrosis (MF) is a chronic and progressive myeloproliferative neoplasm accompanied by symptoms including, splenomegaly, fatigue, night sweats, fever, pruritis and anemia, and the treatment of myelofbrosis-associated anemia remains suboptimal, with no improved therapies.

Method We retrospectively collected MF with refractory anemia with luspatercept.

Result32 patients with MF treated with luspatercept were enrolled. Median age was 59 years (range, 48-83 years), and 62.5% were males. 28 (87.5%) of patients with hemoglobin below 60g/L. All patients previously were treated with a PTD regimen (prednisone, danazol, thalidomide). 20 (62.5%) were classified as intermediate-1 risk, and 12 (37.5%) as intermediate-2 risk based on Dynamic International Prognostic Scoring System (DIPSS). The median follow-up was 7.5 (3-16) months. 28 (87.5%) patients received luspatercept 50mg every three weeks, of these, 2 patients increased the dose due to poor response; and another 4 patients received luspatercept 25mg every three weeks. Erythroid response occurred in 18 patients (56.3%) at week 12, another 6 patients (18.8%) at week 24, and 25 (78.1%) at the end of follow-up. Patients had a significantly higher hemoglobin level at 12 weeks, 24 weeks, and at the end of follow-up, than at baseline (P=0.002, P=0.005, and P=0.01, respectively). Treatment-related adverse events occurred in 5 (15.6%) patients, with no serious adverse events. 3 (9.4%) patients treatment failure, and 4 (12.5%) patients relapse at week 21 and 24. No patient progressed to acute myeloid leukemia. No patients died by the end of follow-up.

Conclusion In patients with myelofibrosis, luspatercept improved anemia with a low relapse rate and good tolerance.

Keyword Luspatercept; refractory anemia; myelofbrosis

Disclosures

No relevant conflicts of interest to declare.

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2024
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