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Clin Cancer Res. 2022 Sep 15;28(18):3940-3949. doi: 10.1158/1078-0432.CCR-22-0560.
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4
Comprehensive review of statistical methods for analysing patient-reported outcomes (PROs) used as primary outcomes in randomised controlled trials (RCTs) published by the UK's (HTA) journal (1997-2020).对英国 HTA 期刊（1997-2020 年）发表的作为主要结局指标的随机对照试验（RCTs）中使用的患者报告结局（PRO）的统计方法进行全面回顾。
BMJ Open. 2021 Sep 6;11(9):e051673. doi: 10.1136/bmjopen-2021-051673.
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终点选择和评估在血液学研究中的应用。

Endpoint selection and evaluation in hematology studies.

机构信息

Division of Biostatistics, Institute for Health and Equity, Medical College of Wisconsin, 8701 Watertown Plank Road, Milwaukee, WI, 53226, USA.

Division of Hematology and Oncology, Department of Medicine, Medical College of Wisconsin, 8701 Watertown Plank Road, Milwaukee, WI, 53226, USA.

出版信息

Best Pract Res Clin Haematol. 2023 Sep;36(3):101479. doi: 10.1016/j.beha.2023.101479. Epub 2023 May 24.

DOI:10.1016/j.beha.2023.101479
PMID:37611997
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10979628/
Abstract

Observational studies and clinical trials in hematology aim to examine treatments for blood disorders. The outcomes being studied must address the goals of the study and provide meaningful information about treatment course, disease progression, describe patients' survival experience and quality of life. Endpoints are the specific measures of these outcomes, and much consideration should be given to their selection. In this review, we describe the outcomes and endpoints frequently used in studying hematologic diseases and provide general guidelines for their statistical analysis. The main focus is on clinical outcomes which are commonly used in establishing treatment safety and efficacy. We also briefly discuss the role surrogate and composite endpoints play in hematology studies. The importance of patient reported outcomes to comprehensive assessment of the treatment effectiveness is highlighted. Provided practical considerations for choosing primary and secondary endpoints may be helpful in designing hematology clinical trials.

摘要

观察性研究和血液病临床试验旨在检查血液疾病的治疗方法。正在研究的结果必须针对研究目标，并提供有关治疗过程、疾病进展、描述患者生存体验和生活质量的有意义信息。终点是这些结果的具体衡量指标，应充分考虑其选择。在这篇综述中，我们描述了研究血液病时常用的结果和终点，并提供了其统计分析的一般准则。主要重点是常用于确定治疗安全性和疗效的临床结果。我们还简要讨论了替代终点和复合终点在血液学研究中的作用。强调了患者报告结果对治疗效果综合评估的重要性。选择主要和次要终点的实际考虑因素可能有助于设计血液学临床试验。