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Study to Evaluate the Long- Term Safety and Efficacy of Luspatercept in Subjects Who Received at Least One Dose of Luspatercept in the Compassionate Use Phase

Sponsor: Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia

Summary

Luspatercept represents the first and only erythroid maturation agent (EMA) approved by the European Commission (EC) and the Food and Drug Administration (FDA) capable of enhancing advanced erythrocyte maturation. The efficacy of luspatercept was demonstrated in the phase III clinical trial called "BELIEVE." More than 200 Italian patients with transfusion- dependent beta thalassemia aged ≥18 years who had no approved therapeutic alternatives to improve their clinical course were considered eligible for the 'compassionate' use program related to luspatercept and most of them received at least one dose of the drug before it was dispensed by the National Health System (NHS), after approval by the pharmaceutical company and the Ethics Committee of the Clinical Center in which they were being followed.

Official title: Observational Study to Evaluate the Long- Term Safety and Efficacy of Luspatercept in Subjects Who Received at Least One Dose of Luspatercept in the "Compassionate" Use Phase

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