Causal inference in a clinical trial: A comparative example

被引:23
作者
Heitjan, DF [1 ]
机构
[1] Columbia Univ, Joseph L Mailman Sch Publ Hlth, Div Biostat, New York, NY 10032 USA
来源
CONTROLLED CLINICAL TRIALS | 1999年 / 20卷 / 04期
关键词
Intention-to-treat analysis; noncompliance; nonignorable model;
D O I
10.1016/S0197-2456(99)00012-4
中图分类号
R-3 [医学研究方法]; R3 [基础医学];
学科分类号
1001 ;
摘要
Recently there has been much interest in methods for analyzing clinical trials of treatments that are subject to noncompliance. In this paper I study a small, simple dataset from a clinical trial of immunosuppressive therapy in the treatment of multiple sclerosis. I apply and compare a range of methods: the as-randomized (intention-to-treat) analysis, the as-treated analysis, estimates based on a nonignorable selection model, and Rubin's causal model. The results differ substantially even in this small dataset that exhibits modest noncompliance. For this reason, data analysts should be clear about which parameters are of greatest importance in the analysis of a clinical trial. Control Clin Trials 1999;20:309-318 (C) Elsevier Science Inc. 1999.
引用
收藏
页码:309 / 318
页数:10
相关论文
共 12 条
[1]  
Angrist JD, 1996, J AM STAT ASSOC, V91, P444, DOI 10.2307/2291629
[2]  
CRITCHLEY F, 1988, BIOMETRIKA, V75, P21
[3]  
Diggle P. G., 1994, J ROY STAT SOC C, V43, P49
[4]  
EFRON B, 1991, J AM STAT ASSOC, V86, P9, DOI 10.2307/2289707
[5]   A PLACEBO-CONTROLLED, RANDOMIZED, DOUBLE-MASKED, VARIABLE DOSAGE, CLINICAL-TRIAL OF AZATHIOPRINE WITH AND WITHOUT METHYLPREDNISOLONE IN MULTIPLE-SCLEROSIS [J].
ELLISON, GW ;
MYERS, LW ;
MICKEY, MR ;
GRAVES, MC ;
TOURTELLOTTE, WW ;
SYNDULKO, K ;
HOLEVOETHOWSON, MI ;
LERNER, CD ;
FRANE, MV ;
PETTLERJENNINGS, P .
NEUROLOGY, 1989, 39 (08) :1018-1026
[7]  
HEITJAN DF, 1999, IN PRESS STAT MED
[8]  
Imbens GW, 1997, ANN STAT, V25, P305
[9]   INTENTION-TO-TREAT ANALYSIS AND THE GOALS OF CLINICAL-TRIALS [J].
SHEINER, LB ;
RUBIN, DB .
CLINICAL PHARMACOLOGY & THERAPEUTICS, 1995, 57 (01) :6-15
[10]  
SOMMER A, 1994, STAT MED, V13, P1897