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

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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Publication Details

Journal
Mediterranean Journal of Hematology and Infectious Diseases
Published
2025-08-31
DOI
https://doi.org/10.4084/mjhid.2025.059
Primary Topic
Hemoglobinopathies and Related Disorders
Type
article
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article

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

Huang Jian, Yu Tang, Xiaolin Yin, Changyu Yang et al.
Mediterranean Journal of Hematology and Infectious Diseases
Hemoglobinopathies and Related Disorders
article

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

Huang Jian, Yu Tang, Xiaolin Yin, Changyu Yang, Guiping Liao, Yali Zhou, Dongmei Liu, Beibei Yang, Yingying Li
article en

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.

Mediterranean Journal of Hematology and Infectious DiseasesVol. 17(1)
303 Hospital of People's Liberation Army (CN)
Openalex Percentile: Top 68%
Hemoglobinopathies and Related Disorders
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