Objectives: Erythropoiesis-stimulating agents (ESA) have an established role in treating anemia in hematological malignancies. However, their role, particularly biosimilar ESA (B-ESA), in myelofibrosis (MF) is not well established.
Methods: This study retrospectively collected data on 96 MF patients treated with B-ESA (alpha/zeta) for the management of anemia to assess safety, efficacy (anemia response [AR]), and survival.
Results: Seventy-seven patients (80%) obtained AR. The median time to AR was 2.5 months. In multivariate analysis, significant predictive factors of AR were transfusion independency (p = .006) and ferritin levels <200 ng/ml (p = .009) at baseline. After a median follow-up of 43.8 months from diagnosis, 38 patients (39%) died, 11 (28.9%) from leukemic evolution. Only two patients (2.5%) stopped B-ESA for toxicity. The 24-month survival was significantly affected by response to B-ESA (70.8% in AR vs. 55.3% in non-responder patients, p = .016). In multivariate analysis, age ≤ 70 years (p = .029) and Hb > 8.5 g/dl (p = .047) at baseline were significantly associated with improved survival, with a trend for longer survival in AR patients (p = .06).
Conclusions: B-ESA seems to be an effective and well-tolerated option for anemia treatment in the MF setting. This strategy deserves further clinical investigation.
Keywords: Janus kinase inhibitors; anemia; biosimilar pharmaceuticals; erythropoiesis-stimulating agents; myelofibrosis; outcome.
© 2022 The Authors. European Journal of Haematology published by John Wiley & Sons Ltd.