Thalidomide for Recurrent Bleeding Due to Small-Intestinal Angiodysplasia

作者：Huimin Chen, Shan Wu, Mingyu Tang, Ran Zhao, Qingwei Zhang, Zihao Dai, Yun–Jie Gao, Shiming Yang, Zhao‐Shen Li, Yiqi Du, Aiming Yang, Liang Zhong, Lungen Lu, Leiming Xu, Xizhong Shen, Side Liu, Jie Zhong, Xiaobo Li, Hong Lü, Hua Xiong, Yufeng Shen, Haiying Chen, Shuai Gong, Hanbing Xue, Zhi‐Zheng Ge · 发表于：New England Journal of Medicine · 年份：2023 · DOI：10.1056/nejmoa2303706 · 被引用次数：70 · 研究领域：Gastrointestinal Bleeding Diagnosis and Treatment、Abdominal Trauma and Injuries、Platelet Disorders and Treatments

BACKGROUND: Recurrent bleeding from the small intestine accounts for 5 to 10% of cases of gastrointestinal bleeding and remains a therapeutic challenge. Thalidomide has been evaluated for the treatment of recurrent bleeding due to small-intestinal angiodysplasia (SIA), but confirmatory trials are lacking. METHODS: We conducted a multicenter, double-blind, randomized, placebo-controlled trial to investigate the efficacy and safety of thalidomide for the treatment of recurrent bleeding due to SIA. Eligible patients with recurrent bleeding (at least four episodes of bleeding during the previous year) due to SIA were randomly assigned to receive thalidomide at an oral daily dose of 100 mg or 50 mg or placebo for 4 months. Patients were followed for at least 1 year after the end of the 4-month treatment period. The primary end point was effective response, which was defined as a reduction of at least 50% in the number of bleeding episodes that occurred during the year after the end of thalidomide treatment as compared with the number that occurred during the year before treatment. Key secondary end points were cessation of bleeding without rebleeding, blood transfusion, hospitalization because of bleeding, duration of bleeding, and hemoglobin levels. RESULTS: Overall, 150 patients underwent randomization: 51 to the 100-mg thalidomide group, 49 to the 50-mg thalidomide group, and 50 to the placebo group. The percentages of patients with an effective response in the 100-mg thalidomi...