What question did this study set out to answer?

To quantify the efficacy and safety of luspatercept in β-thalassemia across different patient populations.

December 8, 2025BloodOpen Access

Luspatercept in β-thalassemia: Meta-analysis of efficacy and safety across transfusion-dependent and non-transfusion-dependent populations

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Meta-analysis finds luspatercept significantly reduces transfusion needs in β-thalassemia, indicating strong efficacy and safety.

To quantify the efficacy and safety of luspatercept in β-thalassemia across different patient populations.
Conducted a meta-analysis using prospective trials of luspatercept in β-thalassemia.
Included data from four studies with a total of 393 patients, involving randomized and phase II trials.
Pooled results for transfusion reduction and hemoglobin increase were calculated using random-effects models.
In transfusion-dependent β-thalassemia, 74% achieved ≥33% reduction in transfusion burden, 27% achieved ≥50% reduction.
In non-transfusion-dependent β-thalassemia, 78% achieved ≥1.0 g/dL hemoglobin increase, 54% achieved ≥1.5 g/dL increase.
Overall, 98% of patients experienced any treatment-emergent adverse events with an acceptable safety profile.

Nimmagadda et al. (2025) studied this question.

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December 8, 2025BloodOpen Access

View Full Paper

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Authors

Meta-analysis finds luspatercept significantly reduces transfusion needs in β-thalassemia, indicating strong efficacy and safety.

To quantify the efficacy and safety of luspatercept in β-thalassemia across different patient populations.
Conducted a meta-analysis using prospective trials of luspatercept in β-thalassemia.
Included data from four studies with a total of 393 patients, involving randomized and phase II trials.
Pooled results for transfusion reduction and hemoglobin increase were calculated using random-effects models.
In transfusion-dependent β-thalassemia, 74% achieved ≥33% reduction in transfusion burden, 27% achieved ≥50% reduction.
In non-transfusion-dependent β-thalassemia, 78% achieved ≥1.0 g/dL hemoglobin increase, 54% achieved ≥1.5 g/dL increase.
Overall, 98% of patients experienced any treatment-emergent adverse events with an acceptable safety profile.

Nimmagadda et al. (2025) studied this question.

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