中华眼底病杂志

中华眼底病杂志

糖尿病视网膜病变临床研究设计的思考:从随机对照试验到真实世界研究

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糖尿病视网膜病变(DR)是糖尿病患者的常见并发症,致盲率高,在给社会带来经济负担的同时,严重威胁患者的生活质量。围绕DR治疗效果的临床研究是当前的热点领域,但在开展临床研究之前,我们应当首先做好科学的研究设计。随机对照试验(RCT)被认为是循证医学的金标准,但其同样存在局限性,研究结论应用于实际临床工作之前仍需客观辩证的解读。真实世界研究(RWS)可弥合RCT与临床实践的距离,同时具有数据易于获取、节省资源和时间等优势,能够较快得出结果。但RWS也面临无标准化数据源和易受混杂偏倚影响的挑战。在DR的RWS中,标准化单病种数据库能够提供广泛的数据来源,倾向性评分匹配方法能够降低混杂偏倚的影响。

Diabetic retinopathy (DR), which is a common complication of diabetic and the main cause of blindness, brings not only a heavy economic burden to society, but also seriously threatens to the patients’ quality of life. Clinical researches on the therapies of DR are active at present, but how to perform a good clinical research with scientific design should be considered with high priority. The randomized controlled trial (RCT) is considered to be the gold standard for evidence-based medicine, but RCT is not always perfect. Limitations still exist in certain circumstance and the conclusions from RCTs also need to be interpreted by an objective point of view before clinical practice. Real world study (RWS) bridges the gap between RCT and clinical practice, in which the data can be easily collected without much cost, and results might be obtained within a short period. However, RWS is also faced with the challenge of not having standardized data and being susceptible to confounding bias. The standardized single disease database for DR and propensity score matching method can provide a wide range of data sources and avoid of bias for RWS in DR.

关键词: 糖尿病视网膜病变; 随机对照试验; 倾向性评分; 述评; 真实世界研究

Key words: Diabetic retinopathy; Randomized controlled trial; Propensity score; Editorial; Real-world study

引用本文: 孙晓东, 贾慧珣. 糖尿病视网膜病变临床研究设计的思考:从随机对照试验到真实世界研究. 中华眼底病杂志, 2019, 35(2): 111-114. doi: 10.3760/cma.j.issn.1005-1015.2019.02.001 复制

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