Design and analysis of randomized clinical trials requiring prolonged observation of each patient. I. Introduction and design.
Design and analysis of randomized clinical trials requiring prolonged observation of each patient. I. Introduction and design.
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DOI：
10.1038/bjc.1976.220
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发表时间：
1976-12
影响因子：
8.8
通讯作者：
Smith, P G
中科院分区：
文献类型：
--
作者：
Peto, R;Pike, M C;Armitage, P;Breslow, N E;Cox, D R;Howard, S V;Mantel, N;McPherson, K;Peto, J;Smith, P G
医学研究理事会多年来一直鼓励在白血病和其他癌症方面进行合作临床试验，并在医学文献中报告试验结果。值得发表的一项未报告的成果是发展了设计和分析这类试验的专门知识。这份报告是由一群英国和美国的统计学家编写的，但它是为没有任何统计专业知识的人准备的。第一部分，出现在这个问题上，讨论了这样的试验的设计;第二部分，将单独出现在1977年1月的杂志上，通过寿命表和对数秩检验，包括一个工作的例子，并讨论了试验结果的解释，包括2个特定试验的简要报告，给出了完整的说明。本报告的两个部分均与研究至死亡时间的所有临床试验相关，并且将与研究至其他特定类别的不良事件的时间的临床试验同样相关：首次卒中，可能，或首次复发，转移，疾病复发，血栓形成，移植排斥，或特定原因的死亡。本期的第一部分收集了医学文献中已经出现的观点，但下个月的第二部分是为非统计学医生出版的第一个关于如何有效分析生存期临床试验数据的简单说明。这些试验包括大多数癌症治疗的临床试验;然而，在癌症试验中，除了研究总生存期外，最好使用这些统计方法来研究肿瘤局部复发的时间，或研究可检测的转移性扩散的时间。实体瘤可以在诊断时进行分期;如果这一点或其他疾病的任何其他可用信息是结局的重要决定因素，则可以使用它来使整个异质性试验人群的总体对数秩检验更加敏感，并且在直观上更加令人满意，因为这样就只需要进行同类比较，而不是偶然地将I期与III期进行比较。
The Medical Research Council has for some years encouraged collaborative clinical trials in leukaemia and other cancers, reporting the results in the medical literature. One unreported result which deserves such publication is the development of the expertise to design and analyse such trials. This report was prepared by a group of British and American statisticians, but it is intended for people without any statistical expertise. Part I, which appears in this issue, discusses the design of such trials; Part II, which will appear separately in the January 1977 issue of the Journal, gives full instructions for the statistical analysis of such trials by means of life tables and the logrank test, including a worked example, and discusses the interpretation of trial results, including brief reports of 2 particular trials. Both parts of this report are relevant to all clinical trials which study time to death, and wound be equally relevant to clinical trials which study time to other particular classes of untoward event: first stroke, perhaps, or first relapse, metastasis, disease recurrence, thrombosis, transplant rejection, or death from a particular cause. Part I, in this issue, collects together ideas that have mostly already appeared in the medical literature, but Part II, next month, is the first simple account yet published for non-statistical physicians of how to analyse efficiently data from clinical trials of survival duration. Such trials include the majority of all clinical trials of cancer therapy; in cancer trials,however, it may be preferable to use these statistical methods to study time to local recurrence of tumour, or to study time to detectable metastatic spread, in addition to studying total survival. Solid tumours can be staged at diagnosis; if this, or any other available information in some other disease is an important determinant of outcome, it can be used to make the overall logrank test for the whole heterogeneous trial population more sensitive, and more intuitively satisfactory, for it will then only be necessary to compare like with like, and not, by chance, Stage I with Stage III.