1. Luspatercept in the Treatment of Beta Thalassemia in Italy: Lights and Shadows in Clinical Practice
- Author
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Gianesin, Barbara, Rosso, Rosamaria, Lisi, Roberto, Zappu, Antonietta, Motta, Irene, Longo, Filomena, Pinto, Valeria, Sanna, Paola Maria Grazia, De Franceschi, Lucia, Ruffo, Giovan Battista, DI Maggio, Rosario, Bulla, Anna, Di Giorgio, Mary Ann, Denotti, Anna Rita, Panzieri, Daniele Lello, Culcasi, Martina, Quintino, Sabrina, Marchisio, Andrea, Mazzi, Filippo, Miciotto, Francesca, Barone, Rita, Di Raimondo, Francesco, Ferraresi, Marta, Marchetti, Beatrice, and Origa, Raffaella
- Abstract
Luspatercept has been demonstrated to reduce the need for blood transfusion with manageable side effects in clinical trials, thus receiving the approval for the treatment of anemia associated with transfusion dependent beta thalassemia in adults. However, very little data on its safety and efficacy in real life have been hitherto published. Special warnings and precautions for use refer to the possibility of occurrence of thromboembolic events, extramedullary hemopoiesis masses and increased blood pressure. Ten Italian Specialized Centres for the treatment of Hemoglobinopathies, with the patronage of the “Società Italiana Talassemie ed Emoglobinopatie” (SITE), evaluated the effects of luspatercept in terms of safety and blood transfusion reduction in 116 patients (Table 1) who received the first dose of the drug after its marketing. The median duration of treatment was 158 days (min-max: 15-442). β globin genotype was β 0/β 0in 42 subjects (36.2%), β 0/ β +in 33 (28.4%), β +/ β +in 23 (19.8%), β 0/β wtplus α gene triplication in 4 (3.4%) and unknown in 14 (12%). Sixty-four patients (55.2%) were splenectomized. At the time of data collection, 32 patients (27.6%) had already discontinued the drug. Discontinuation had occurred after a median time of 132 days of treatment (min-max: 15-338) due to insufficient efficacy in the judgement of the physician (10) or patient (3), side effects attributed to luspatercept (16) that were detrimental to the continuation of therapy in the judgement of the physician (6) or patient (10), pregnancy (1) and other causes (2).
- Published
- 2023
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