Luspatercept for Transfusion-Dependent Beta-Thalassemia: Real-World Experience in a Large Cohort of Patients From Italy

The randomized, placebo‐controlled phase III BELIEVE study led to the approval of luspatercept to promote erythroid maturation in the United States and Europe [1]. Given the need for scientific evidence on its efficacy, tolerability, and safety in clinical practice, we evaluated the effects of luspatercept in 231 patients with transfusion‐dependent thalassemia (TDT) (Figure S1) who received their first dose of the drug post‐marketing at 27 Italian specialized centers under the patronage of the Società Italiana Talassemia ed Emoglobinopatie (Tables S1 and S2). The median treatment duration was 272 days (Q1–Q3: 150–531, range: 21–1007). At the time of data collection, 106 patients (45.9%) had discontinued the drug after a median time of 172 days of treatment (Q1–Q3: 99–307, range: 21–671) (Figures S2 and S3, Table S3). In part, the high number of patients who prematurely discontinued may be associated with the fact that the lives of these patients revolve around transfusions and the transfusion cycle governs every aspect of their existence. Consequently, the loss of a normal transfusion schedule and/or transfusion independence can create anxiety and insecurity. A solid doctor‐patient therapeutic alliance is essential to begin therapy under optimal conditions for success. In our study, both the primary and secondary endpoints of the BELIEVE trial were achieved at comparable or significantly higher rates. During the treatment period, 44 patients (19%) had a transfusion‐free interval of at least 8 weeks (median: 14.9 weeks, range: 8–115 weeks).