• Huimin Chen, Shan Wu, Mingyu Tang, Ran Zhao, Qingwei Zhang, Zihao Dai, Yunjie Gao, Shiming Yang, Zhaoshen Li, Yiqi Du, Aiming Yang, Liang Zhong, Lungen Lu, Leiming Xu, Xizhong Shen, Side Liu, Jie Zhong, Xiaobo Li, Hong Lu, Hua Xiong, Yufeng Shen, Haiying Chen, Shuai Gong, Hanbing Xue, and Zhizheng Ge.
• From the Division of Gastroenterology and Hepatology, Shanghai Jiao-Tong University School of Medicine Renji Hospital, Shanghai Institute of Digestive Disease, Key Laboratory of Gastroenterology and Hepatology, Ministry of Health (Shanghai Jiao-Tong University) (Huimin Chen, S.W., M.T., R.Z., Q.Z., Z.D., Y.G., X.L., H.L., H. Xiong, Y.S., Haiying Chen, S.G., H. Xue, Z.G.), the Department of Gastroenterology and Hepatology, Changhai Hospital, Naval Medical University (Z.L., Y.D.), the Department of Gastroenterology, Huashan Hospital (L.Z.), and the Department of Gastroenterology and Hepatology, Zhongshan Hospital (X.S.), Fudan University, the Department of Gastroenterology, Shanghai General Hospital, Shanghai Jiao-Tong University School of Medicine (L.L.), the Department of Gastroenterology, Xinhua Hospital, Shanghai Jiao-Tong University School of Medicine (L.X.), and the Department of Gastroenterology, Ruijin Hospital, Shanghai Jiao-Tong University School of Medicine (J.Z.), Shanghai, the Department of Gastroenterology, Xinqiao Hospital, Army Medical University, Chongqing (S.Y.), the Department of Gastroenterology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing (A.Y.), and the Department of Gastroenterology, Guangdong Provincial Key Laboratory of Gastroenterology, Nanfang Hospital, Southern Medical University, Guangzhou (S.L.) - all in China.
• N. Engl. J. Med. 2023 Nov 2; 389 (18): 164916591649-1659.

BackgroundRecurrent 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.MethodsWe 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.ResultsOverall, 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 thalidomide group, 50-mg thalidomide group, and placebo group were 68.6%, 51.0%, and 16.0%, respectively (P<0.001 for simultaneous comparison across the three groups). The results of the analyses of the secondary end points supported those of the primary end point. Adverse events were more common in the thalidomide groups than in the placebo group overall; specific events included constipation, somnolence, limb numbness, peripheral edema, dizziness, and elevated liver-enzyme levels.ConclusionsIn this placebo-controlled trial, treatment with thalidomide resulted in a reduction in bleeding in patients with recurrent bleeding due to SIA. (Funded by the National Natural Science Foundation of China and the Shanghai Municipal Education Commission, Gaofeng Clinical Medicine; ClinicalTrials.gov number, NCT02707484.).Copyright © 2023 Massachusetts Medical Society.

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