Can I read Long-term Safety and Erythroid Response with Luspatercept Treatment in Patients with β-Thalassemia on EtoBox?

Long-term Safety and Erythroid Response with Luspatercept Treatment in Patients with β-Thalassemia by Antonio Piga; Filomena Longo; Maria Rita Gamberini; Ersi Voskaridou; Paolo Ricchi; Vincenzo Caruso; Antonello Pietrangelo; Xiaosha Zhang; Jeevan K. Shetty; Kenneth M. Attie; Immacolata Tartaglione is a Medicine article available to read on EtoBox.

What is Long-term Safety and Erythroid Response with Luspatercept Treatment in Patients with β-Thalassemia about?

## Background: β-thalassemia is a hereditary blood disorder resulting in ineffective erythropoiesis and anemia. Management of anemia with regular blood transfusions is associated with complications including iron overload. Here, we report long-term safety and efficacy results of the first clinical study of luspatercept in β-thalassemia, initiated in 2013, enrolling adults with both nontransfusion-dependent (NTD) and transfusion-dependent (TD) β-thalassemia. ## Objectives: The objective was to report long-term safety data, for up to 5 years of treatment, for 64 patients with TD or NTD β-thalassemia, and long-term efficacy data for a subset of 63 patients with β-thalassemia who received high-dose luspatercept (0.6–1.25 mg/kg): 31 NTD and 32 TD patients. ## Design: The study was a phase 2, noncontrolled, open-label trial comprising a dose-finding base phase and a 5-year extension phase. ## Methods: Endpoints include safety; erythroid response over a continuous 12-week period [NTD: hemoglobin increase from baseline ⩾1.0 or ⩾1.5 g/dl; TD: red blood cell (RBC) transfusion burden reduction, ⩾20%, ⩾33%, or ⩾50%]; and changes in biomarkers of ineffective erythropoiesis, iron metabolism para

Who reads Long-term Safety and Erythroid Response with Luspatercept Treatment in Patients with β-Thalassemia?

Author

Antonio Piga; Filomena Longo; Maria Rita Gamberini; Ersi Voskaridou; Paolo Ricchi; Vincenzo Caruso; Antonello Pietrangelo; Xiaosha Zhang; Jeevan K. Shetty; Kenneth M. Attie; Immacolata Tartaglione

Published

2022

Language

EN

Field

Medicine (Health Sciences)