Scientific Letters
Vol. 17 No. 1 (2025): Review Articles, Original Article, Scientific Letter, Case Reports Letter to the Editor

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

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Received: June 11, 2025
Accepted: July 26, 2025
Published: August 31, 2025
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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.

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Benign Hematology

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“Luspatercept Treatment in a β-Thalassemia Patient with Pulmonary Arterial Hypertension: A Case Report” (2025) Mediterranean Journal of Hematology and Infectious Diseases, 17(1), p. e2025059. doi:10.4084/MJHID.2025.059.