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Luspatercept Treatment in a β-Thalassemia Patient with Pulmonary Arterial Hypertension: A Case Report

Year: 2025

Type: article

Abstract: β-Thalassemia is associated with a range of complications, with pulmonary hypertension in affected individuals having a particularly high mortality rate. However, data on the use of pulmonary vasodilators in β-thalassemia are scarce. Here, we present the case of an adult male diagnosed with β-thalassemia and pulmonary hypertension. There was no improvement in symptoms or test results with low-dose Riociguat. After taking Luspatercept, the patient's hemoglobin level increased significantly. Notably, his PAH symptoms also improved, as reflected by better 6-minute walk test results and reduced tricuspid regurgitation velocity on echocardiography. These findings suggest that Luspatercept may be useful in reducing pulmonary vascular resistance and improving clinical outcomes in patients with β-thalassemia and pulmonary hypertension.">β-Thalassemia is associated with a range of complications, with pulmonary hypertension in affected individuals having a particularly high mortality rate. However, data on the use of pulmonary vasodilators in β-thalassemia are scarce. Here, we present the case of an adult male diagnosed with β-thalassemia and pulmonary hypertension. There was no improvement in symptoms or test results with low-dose Riociguat. After taking Luspatercept, the patient's hemoglobin level increased significantly. Notably, his PAH symptoms also improved, as reflected by better 6-minute walk test results and reduced tricuspid regurgitation velocity on echocardiography. These findings suggest that Luspatercept may be useful in reducing pulmonary vascular resistance and improving clinical outcomes in patients with β-thalassemia and pulmonary hypertension.

Source: Mediterranean Journal of Hematology and Infectious Diseases

DOI: 10.4084/mjhid.2025.059