■胃肠道(GI)血管增生是危及生命的出血的潜在原因。沙利度胺可能具有一定的治疗效果。
■我们旨在评估沙利度胺的疗效和安全性,并使用试验序贯分析(TSA)来评估进一步随机对照试验(RCT)的必要性。
■随机对照试验的Meta分析。
■我们系统地搜索了Cochrane中央对照试验登记册(CENTRAL),医学文献在线分析和检索系统(MEDLINE),Embase,万方,和中国国家知识基础设施数据库,用于评估沙利度胺在无语言限制的胃肠道血管增生中的RCT。我们使用随机效应模型来获取池数据,并遵循建议评估的分级,发展和评价框架。TSA用于控制随机误差的风险并评估我们结论的有效性。
■纳入三项随机对照试验,涉及279例患者,小肠血管增生比例为87.1%。沙利度胺改善血红蛋白水平的平均变化[平均差异(MD):3.06,95%置信区间:2.66-3.46],无严重不良反应发生。其他次要终点,包括有效反应率,治疗后出血停止,因出血住院率,出血住院时间的变化,红细胞输注要求,和总体不利影响,与对照组相比,沙利度胺组的结局也明显更好。所有结果的TSA超出了所需的信息大小,和累积Z曲线所有遍历试验序贯监测边界。
■几乎所有的证据都是中等质量的,这表明沙利度胺有望治疗胃肠道血管增生,具有良好的安全性。TSA建议,建议进行大规模的现实世界研究,而不是仅仅依靠在同一人群和试验设计中进行的随机对照试验。
■该荟萃分析方案已在PROSPERO(CRD42023480621)上注册。
UNASSIGNED: Gastrointestinal (GI) angiodysplasias is a potential cause of life-threatening bleeding.
Thalidomide may have a certain effect on the treatment.
UNASSIGNED: We aim to evaluate the efficacy and safety of
thalidomide and used trial sequential analysis (TSA) to assess the need for further randomized controlled trials (RCTs).
UNASSIGNED: Meta-analysis of RCTs.
UNASSIGNED: We systematically searched Cochrane Central Register of Controlled Trials (CENTRAL), Medical Literature Analysis and Retrieval System Online (MEDLINE), Embase, WanFang, and China National Knowledge Infrastructure databases for RCTs evaluating
thalidomide in GI angiodysplasias without language restrictions. We used a random-effects model to obtain pool data and followed Grading of Recommendations Assessment, Development and Evaluation framework. TSA was employed to control the risk of random errors and to evaluate the validity of our conclusions.
UNASSIGNED: Three RCTs were included involving 279 patients with the proportion of small intestinal angiodysplasias of 87.1%.
Thalidomide led to improved mean change of hemoglobin level [mean difference (MD): 3.06, 95% confidence interval: 2.66-3.46] without severe adverse effects occurring. Other secondary endpoints, including effective response rate, cessation of bleeding after treatment, hospitalization rate because of bleeding, change in duration of hospital stays for bleeding, transfused red cell requirements, and overall adverse effects, also showed significantly better outcomes in the thalidomide group compared to the control group. TSA for all outcomes exceeded required information sizes, and cumulative Z curve all traverse trial sequential monitoring boundary.
UNASSIGNED: Almost all of the evidence was of moderate quality, suggesting that
thalidomide holds promise for treating GI angiodysplasias, with favorable safety profiles. TSA suggests that conducting large-scale real-world research is recommended over relying solely on RCTs conducted within the same population and trial design.
UNASSIGNED: This meta-analysis protocol was registered on PROSPERO (CRD42023480621).