Efficacy and safety of thalidomide in β-thalassaemia: a systematic review and meta-analysis

Thalidomide has shown promise as an adjunct therapy for β-thalassaemia, yet its effectiveness and safety are uncertain. This systematic review and meta-analysis evaluate the efficacy and safety of thalidomide in β-thalassaemia; in both transfusion-dependent and non-transfusion-dependent patients. Scopus, PubMed and Cochrane databases were searched for studies assessing the efficacy and safety of thalidomide in β-thalassaemia. Primary outcomes included transfusion response and haemoglobin increases whereas adverse events and effects on serum ferritin and spleen size were secondary outcomes. Study quality, risk of bias and heterogeneity were assessed using standardized tools. Nineteen studies comprising 1731 patients met the eligibility criteria. Thalidomide halved the transfusion requirements in 76% patients with transfusion-dependent β-thalassaemia (response rate of 0.76 [95%CI: 0.67-0.83]) and achieved transfusion independence in 55% (good response rate of 0.55 [95%CI: 0.47-0.63]). In non-transfusion-dependent β-thalassaemia, > 1 g/dl and > 2 g/dL rise in haemoglobin were seen among 91% (response rate- 0.91 [95%CI: 0.81-0.96]) and 76% (good response rate- 0.76 [95%CI: 0.63-0.85]) of patients, respectively. The most frequent adverse effects were constipation (15% [95% CI: 13.7-17.2]) and somnolence (15% [95%CI: 13.6-17.1%]). Thalidomide appears to be effective and tolerable in β-thalassaemia. It reduced the transfusion requirement in transfusion-dependent β-thalassaemia patients and increased the haemoglobin levels in non-transfusion-dependent β-thalassaemia patients.Systematic review registration: CRD42024627095.