ArticleAnnals of hematology2026
Low- to moderate-dose thalidomide combination rescue after luspatercept failure in transfusion-dependent β-thalassemia: a real-world cohort study.
Article in Annals of hematology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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6 authors.
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Abstract
backgroundA proportion of transfusion-dependent β-thalassemia (TDT) patients show suboptimal or heterogeneous response to luspatercept. We explored a structured rescue approach by adding low- to moderate-dose thalidomide after predefined luspatercept failure in real-world practice.
methodsThis study was a single-center retrospective cohort study. Adults with TDT on luspatercept who met predefined criteria for suboptimal response (failure to achieve ≥ 33% reduction in RBC units over a 12-week window versus each patient's baseline 12-week window, or failure to increase Hb by ≥ 1.0 g/dL under an unchanged transfusion trigger) received thalidomide 25-50 mg once daily while continuing luspatercept. Primary endpoint was 12-week transfusion independence (TI), defined as no transfusion for 12 consecutive weeks and Hb ≥ 9.0 g/dL. Secondary endpoints included ≥ 33%/≥50% reductions in RBC units over matched 12-week windows, longitudinal Hb change, and safety graded by CTCAE v5.0 and reported as exposure-adjusted incidence rates.
resultsEleven patients were included (median age 23 years; female 45.5%; β⁰/β⁰ 18.2%; prior splenectomy 63.6%). Twelve-week transfusion independence (TI) was achieved in 4/11 patients (36.4%; 95% CI 15.2-64.6). Proportions with ≥ 33% and ≥ 50% reductions in RBC units over matched 12-week windows were 45.5% (95% CI 21.3-72.0) and 45.5% (95% CI 21.3-72.0), respectively. Hemoglobin increased by 4.0 g/dL (Hodges-Lehmann, 95% CI 2.9-5.0; LMM p < 0.001). Total exposure was 112 patient-months; EAIR for any-grade treatment-emergent adverse events was 8.0 per 100 patient-months. No ≥Grade 2 neurotoxicity or thrombosis occurred.
conclusionsIn TDT patients with predefined luspatercept failure, adding low- to moderate-dose thalidomide was associated with improvements in hemoglobin levels and reductions in transfusion burden with favorable tolerability. These findings warrant prospective validation of this structured rescue approach.
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