Role of thalidomide in angiodysplasia-related gastrointestinal bleeding: a systematic review

Objective Angiodysplasia of the gastrointestinal (GI) tract is a leading cause of occult GI bleeding, and its management remains challenging. Various pharmacologic and endoscopic therapies are used with limited success. This systematic review evaluates the clinical efficacy of thalidomide in angiodysplasia-related GI bleeding. Methods A comprehensive literature search was conducted across PubMed, Embase, Scopus, and CINAHL using MeSH terms “Vascular malformations” OR “Angiodysplasia” AND “Thalidomide,” covering the period from 1994 to February 16, 2024. We included clinical trials and case series with at least five adult patients treated with thalidomide for angiodysplasia-related GI bleeding. Six studies met the inclusion criteria: two randomized controlled trials (RCTs), one retrospective observational study, and three case series. Results A total of 265 patients were included, with a median age of 63.5 years; 37% were male. Angiodysplasia was diagnosed using endoscopy, colonoscopy, or push enteroscopy. Clinical outcomes varied across studies. Garrido et al. reported an 84% response rate based on hemoglobin improvement. In an RCT, Chen et al. demonstrated reduced bleeding episodes in 68.6% of patients receiving thalidomide 100 mg compared with 51% in the 50 mg group. Ge et al. reported a response rate of 71.4% (20/28) in the thalidomide group versus 3.7% (1/27) in the iron group (risk difference 67.7%, 95% CI 51.1–84.2). Common adverse effects included constipation, dizziness, fatigue, limb numbness, and peripheral neuropathy. Conclusion Thalidomide appears effective in reducing bleeding episodes in angiodysplasia-related GI bleeding. However, heterogeneity in dosing, outcome definitions, and safety reporting highlights the need for larger, standardized trials to clarify optimal treatment strategies and long-term safety.