仁济医院NEJM:沙利度胺治疗复发性小肠发育不良出血疗效显著
创作:九卿臣 审核:九卿臣 12月01日
  • 开展一项多中心、双盲、随机、安慰剂对照试验,共150名小肠血管发育不良(SIA)反复出血患者被分配接受沙利度胺或安慰剂治疗4个月,评估沙利度胺对SIA反复出血的疗效和安全性;
  • 其中,51人和49人被随机分配到100 mg和50 mg沙利度胺组,50人被分配到安慰剂组;
  • 100 mg和50 mg沙利度胺组及安慰剂组有效缓解率为68.6%、51.0%和16.0%;
  • 沙利度胺组不良事件发生率高于安慰剂组,如便秘、嗜睡、肢体麻木、外周水肿、头晕和肝酶水平升高。
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九卿臣
小肠反复出血占消化道出血病例的5%~10%,目前仍是治疗难题。先前已有研究评估应用沙利度胺治疗小肠血管发育不良(SIA)引起的反复出血,但仍缺乏确证试验。近日,上海交通大学医学院附属仁济医院消化科戈之铮、陈慧敏及团队在New England Journal of Medicine发表最新研究,开展了一项前瞻性、多中心、随机、双盲、安慰剂对照临床研究,评估沙利度胺对SIA反复出血的疗效和安全性,发现沙利度胺减少了SIA反复出血患者的出血,其效果显著,值得关注。
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Thalidomide for Recurrent Bleeding Due to Small-Intestinal Angiodysplasia

沙利度胺治疗小肠血管发育不良所致复发性出血

10.1056/NEJMoa2303706

11-02, Article

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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 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.
CONCLUSIONS: In 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. opens in new tab.)

First Authors:
Huimin Chen,Shan Wu

Correspondence Authors:
Huimin Chen,Zhizheng Ge

All Authors:
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,Zhizheng Ge

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