Is inverse probability of censoring weighting a safer choice than per-protocol analysis in clinical trials?

被引:0
|
作者
Xuan, Jingyi [1 ]
Mt-Isa, Shahrul [2 ]
Latimer, Nicholas [3 ,4 ]
Gorrod, Helen Bell [3 ]
Malbecq, William [5 ,8 ]
Vandormael, Kristel [6 ]
Yorke-Edwards, Victoria [1 ,7 ]
White, Ian R. [1 ]
机构
[1] UCL, MRC Clin Trials Unit UCL, London, England
[2] MSD, Biostat & Res Decis Sci BARDS, Hlth Technol Assessment HTA Stat, Zurich, Switzerland
[3] Univ Sheffield, Sheffield Ctr Hlth & Related Res, Sch Med & Populat Hlth, Sheffield, England
[4] Delta Hat Ltd, Nottingham, England
[5] Univ Brussels, Dept Math, Brussels, Belgium
[6] MSD, Biostat & Res Decis Sci BARDS, Hlth Technol Assessment HTA Stat, Brussels, Belgium
[7] UCL, Ctr Adv Res Comp, London, England
[8] MSD, Brussels, Belgium
基金
英国医学研究理事会;
关键词
Inverse probability of censoring weight; per-protocol; non-adherence; treatment switching; dependent censoring; MARGINAL STRUCTURAL MODELS; DIRECTED ACYCLIC GRAPHS; 2ND-LINE TREATMENT; NONCOMPLIANCE; AIDS; INTENTION; SURVIVAL; THERAPY; ISSUES; TREAT;
D O I
10.1177/09622802241289559
中图分类号
R19 [保健组织与事业(卫生事业管理)];
学科分类号
摘要
Deviation from the treatment strategy under investigation occurs in many clinical trials. We term this intervention deviation. Per-protocol analyses are widely adopted to estimate a hypothetical estimand without the occurrence of intervention deviation. Per-protocol by censoring is prone to selection bias when intervention deviation is associated with time-varying confounders that also influence counterfactual outcomes. This can be corrected by inverse probability of censoring weighting, which gives extra weight to uncensored individuals who had similar prognostic characteristics to censored individuals. Such weights are computed by modelling selected covariates. Inverse probability of censoring weighting relies on the no unmeasured confounding assumption whose plausibility is not statistically testable. Suboptimal implementation of inverse probability of censoring weighting which violates the assumption will lead to bias. In a simulation study, we evaluated the performance of per-protocol and inverse probability of censoring weighting with different implementations to explore whether inverse probability of censoring weighting is a safe alternative to per-protocol. Scenarios were designed to vary intervention deviation in one or both arms with different prevalences, correlation between two confounders, effect of each confounder, and sample size. Results show that inverse probability of censoring weighting with different combinations of covariates outperforms per-protocol in most scenarios, except for an unusual case where selection bias caused by two confounders is in two directions, and 'cancels' out.
引用
收藏
页码:286 / 306
页数:21
相关论文
共 45 条
  • [31] Prospective Inverse Probability of Treatment-Weighting Analysis of the Clinical Outcome of Red Blood Cell Transfusion Practice in Critically Ill Children
    Singanamalla Bhanudeep
    Ramachandran Rameshkumar
    Muthu Chidambaram
    Tamil Selvan
    Subramanian Mahadevan
    Indian Journal of Pediatrics, 2021, 88 : 985 - 990
  • [32] Intention-to-treat analysis may be more conservative than per protocol analysis in antibiotic non-inferiority trials: a systematic review
    Anthony D. Bai
    Adam S. Komorowski
    Carson K. L. Lo
    Pranav Tandon
    Xena X. Li
    Vaibhav Mokashi
    Anna Cvetkovic
    Aidan Findlater
    Laurel Liang
    George Tomlinson
    Mark Loeb
    Dominik Mertz
    BMC Medical Research Methodology, 21
  • [33] Intention-to-treat analysis may be more conservative than per protocol analysis in antibiotic non-inferiority trials: a systematic review
    Bai, Anthony D.
    Komorowski, Adam S.
    Lo, Carson K. L.
    Tandon, Pranav
    Li, Xena X.
    Mokashi, Vaibhav
    Cvetkovic, Anna
    Findlater, Aidan
    Liang, Laurel
    Tomlinson, George
    Loeb, Mark
    Mertz, Dominik
    BMC MEDICAL RESEARCH METHODOLOGY, 2021, 21 (01)
  • [34] Prospective Inverse Probability of Treatment-Weighting Analysis of the Clinical Outcome of Red Blood Cell Transfusion Practice in Critically Ill Children
    Bhanudeep, Singanamalla
    Rameshkumar, Ramachandran
    Chidambaram, Muthu
    Selvan, Tamil
    Mahadevan, Subramanian
    INDIAN JOURNAL OF PEDIATRICS, 2021, 88 (10): : 985 - 990
  • [35] Effective Treatment of Obstructive Sleep Apnea with CPAP Reduces Major Adverse Cerebrocardiovascular Outcomes: A Systematic Review and Meta-Analysis of Per-Protocol Use of CPAP in Randomized Controlled Trials
    Javaheri, S.
    Martinez-Garcia, M.
    Campos-Rodriguez, F.
    Muriel, A.
    Peker, Y.
    AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE, 2019, 199
  • [36] MABp1 to improve clinical outcomes of patients with symptomatic refractory metastatic colorectal cancer patients: Per-protocol population analysis of phase III study (P1026).
    Wyrwicz, Lucjan
    Saunders, Mark P.
    Andre, Thierry
    Sarosiek, Tomasz
    Nemecek, Radim
    Rogowski, Wojciech
    Leniewski-Kmak, Krzysztof
    Fisher, George A.
    Stecher, Michael
    Mohanty, Prasant
    Simard, John
    Hickish, Tamas
    JOURNAL OF CLINICAL ONCOLOGY, 2017, 35
  • [37] Clinical and cost-effectiveness analysis of mFOLOFX6 with or without a targeted drug among patients with metastatic colorectal cancer: inverse probability of treatment weighting
    Tsai, Hsiang-Lin
    Shi, Hon-Yi
    Chen, Yen-Cheng
    Huang, Ching-Wen
    Su, Wei-Chih
    Chang, Tsung-Kun
    Li, Ching-Chun
    Chen, Po-Jung
    Yeh, Yung-Sung
    Yin, Tzu-Chieh
    Wang, Jaw-Yuan
    AMERICAN JOURNAL OF CANCER RESEARCH, 2023, 13 (09): : 4039 - 4056
  • [38] Use of machine learning to estimate the per-protocol effect of low-dose aspirin on pregnancy outcomes: a secondary analysis of a randomized clinical trial (vol 5, e2143414, 2022)
    Zhong, Y.
    Brooks, M. M.
    Kennedy, E. H.
    Bodnar, L. M.
    Naimi, A., I
    JAMA NETWORK OPEN, 2022, 5 (04)
  • [39] Liver resection versus microwave ablation for hepatocellular carcinoma in ideal candidates for ablation per Barcelona Clinic Liver Cancer staging: a propensity score matching and inverse probability of treatment weighting analysis
    Tong, Yao
    Cai, Ran
    Li, Jun-Xiang
    Chang, De-Hua
    Wang, Li-Zhou
    Cai, Wen-Wu
    Xiao, Yu-Dong
    ALIMENTARY PHARMACOLOGY & THERAPEUTICS, 2022, 56 (11-12) : 1602 - 1614
  • [40] BENEFICIAL EFFECTS OF LUSEOGLIFLOZIN FOR HEPATIC STEATOSIS AND FIBROSIS INDEXES WITH A REDUCTION OF BODY WEIGHT AND AN INCREASE IN SERUM ALBUMIN LEVELS IN PATIENTS WITH DIABETES: A POOLED META- ANALYSIS OF PHASE I I I CLINICAL TRIALS USING INVERSE PROBABILITY TREATMENT WEIGHTING PROPENSITY ANALYSIS
    Kawaguchi, Takumi
    Murotani, Kenta
    Shirouzu, Yoko
    Obara, Hitoshi
    Yamaguchi, Hironori
    Toyofuku, Yuko
    Kaneko, Fumi
    Uchida, Saeko
    HEPATOLOGY, 2023, 78 : S1174 - S1175