[罕见病小样本量临床试验的统计设计与应用]。

Q1 Medicine

中华流行病学杂志 Pub Date : 2024-12-10 DOI:10.3760/cma.j.cn112338-20240705-00401

Y Gao, Z H Zhong, M Y Lu, F Chen

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引用次数: 0

摘要

由于病例数量有限，针对罕见病开展大规模临床试验极具挑战性。本综述介绍了几种为罕见病量身定制的小样本统计设计，包括交叉设计、n-of-1 设计、随机安慰剂阶段设计、随机撤药设计、分组序列设计和自适应设计。报告讨论了这些设计的优缺点和应用场景。此外，还探讨了贝叶斯决策在罕见病临床试验中的优势。本书旨在为罕见病小样本临床试验的设计和实施提供参考。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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[Statistical design and application of clinical trials with small sample sizes for rare diseases].

Due to the limited number of cases, conducting large-scale clinical trials for rare diseases is challenging. This review introduces several small sample statistical designs tailored for rare diseases, including crossover design, n-of-1 design, randomized placebo-phase design, randomized withdrawal design, group sequential design, and adaptive design. It discusses the advantages, disadvantages, and application scenarios of these designs. Additionally, it explores the benefits of Bayes decision-making in clinical trials for rare diseases. The aim is to provide a reference for designing and implementing small sample clinical trials for rare diseases.

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来源期刊

中华流行病学杂志 Medicine-Medicine (all)

CiteScore

5.60

自引率

0.00%

发文量

8981

期刊介绍： Chinese Journal of Epidemiology, established in 1981, is an advanced academic periodical in epidemiology and related disciplines in China, which, according to the principle of integrating theory with practice, mainly reports the major progress in epidemiological research. The columns of the journal include commentary, expert forum, original article, field investigation, disease surveillance, laboratory research, clinical epidemiology, basic theory or method and review, etc. The journal is included by more than ten major biomedical databases and index systems worldwide, such as been indexed in Scopus, PubMed/MEDLINE, PubMed Central (PMC), Europe PubMed Central, Embase, Chemical Abstract, Chinese Science and Technology Paper and Citation Database (CSTPCD), Chinese core journal essentials overview, Chinese Science Citation Database (CSCD) core database, Chinese Biological Medical Disc (CBMdisc), and Chinese Medical Citation Index (CMCI), etc. It is one of the core academic journals and carefully selected core journals in preventive and basic medicine in China.