artcat: Sample-size calculation for an ordered categorical outcome

被引:3
作者
White, Ian R. [1 ]
Marley-Zagar, Ella [1 ]
Morris, Tim P. [1 ]
Parmar, Mahesh K. B. [1 ]
Royston, Patrick [1 ]
Babiker, Abdel G. [1 ]
机构
[1] UCL, MRC Clin Trials Unit, London, England
基金
英国医学研究理事会;
关键词
st0700; artcat; sample size; power; clinical trial; randomized controlled trial; noninferiority trial; substantial-superiority trial; categorical variable; proportional-odds model; evaluation; CLINICAL-TRIALS; SURVIVAL; MULTIARM; EVENTS; POWER;
D O I
10.1177/1536867X231161934
中图分类号
O1 [数学]; C [社会科学总论];
学科分类号
03 ; 0303 ; 0701 ; 070101 ;
摘要
We describe a new command, artcat, that calculates sample size or power for a randomized controlled trial or similar experiment with an ordered categorical outcome, where analysis is by the proportional-odds model. artcat implements the method of Whitehead (1993, Statistics in Medicine 12: 2257-2271). We also propose and implement a new method that 1) allows the user to specify a treatment effect that does not obey the proportional-odds assumption, 2) offers greater accuracy for large treatment effects, and 3) allows for noninferiority trials. We illustrate the command and explore the value of an ordered categorical outcome over a binary outcome in various settings. We show by simulation that the methods perform well and that the new method is more accurate than Whitehead's method.
引用
收藏
页码:3 / 23
页数:21
相关论文
共 15 条
  • [1] Odds ratios should be avoided when events are common
    Altman, DG
    Deeks, JJ
    Sackett, DL
    [J]. BRITISH MEDICAL JOURNAL, 1998, 317 (7168) : 1318 - 1318
  • [2] Evaluation of sample size and power for multi-arm survival trials allowing for non-uniform accrual, non-proportional hazards, loss to follow-up and cross-over
    Barthel, F. M. -S.
    Babiker, A.
    Royston, P.
    Parmar, M. K. B.
    [J]. STATISTICS IN MEDICINE, 2006, 25 (15) : 2521 - 2542
  • [3] A menu-driven facility for complex sample size calculation in randomized controlled trials with a survival or a binary outcome: Update
    Barthel, FMS
    Royston, P
    Babiker, A
    [J]. STATA JOURNAL, 2005, 5 (01) : 123 - 129
  • [4] Anti-influenza hyperimmune intravenous immunoglobulin for adults with influenza A or B infection (FLU-IVIG): a double-blind, randomised, placebo-controlled trial
    Davey, Richard T., Jr.
    Fernandez-Cruz, Eduardo
    Markowitz, Norman
    Pett, Sarah
    Babiker, Abdel G.
    Wentworth, Deborah
    Khurana, Surender
    Engen, Nicole
    Gordin, Fred
    Jain, Mamta K.
    Kan, Virginia
    Polizzotto, Mark N.
    Riska, Paul
    Ruxrungtham, Kiat
    Temesgen, Zelalem
    Lundgren, Jens
    Beigel, John H.
    Lane, H. Clifford
    Neaton, James D.
    [J]. LANCET RESPIRATORY MEDICINE, 2019, 7 (11) : 951 - 963
  • [5] Julious S.A., 2009, Sample sizes for clinical trials, V1st
  • [6] Tutorial in biostatistics - Sample sizes for clinical trials with normal data
    Julious, SA
    [J]. STATISTICS IN MEDICINE, 2004, 23 (12) : 1921 - 1986
  • [7] artbin: Extended sample size for randomized trials with binary outcomes
    Marley-Zagar, Ella
    White, Ian R.
    Royston, Patrick
    Barthel, Friederike M. -S.
    Parmar, Mahesh K. B.
    Babiker, Abdel G.
    [J]. STATA JOURNAL, 2023, 23 (01) : 24 - 52
  • [8] MCCULLAGH P, 1980, J ROY STAT SOC B MET, V42, P109
  • [9] Using simulation studies to evaluate statistical methods
    Morris, Tim P.
    White, Ian R.
    Crowther, Michael J.
    [J]. STATISTICS IN MEDICINE, 2019, 38 (11) : 2074 - 2102
  • [10] A Trial of a Shorter Regimen for Rifampin-Resistant Tuberculosis
    Nunn, Andrew J.
    Phillips, Patrick P. J.
    Meredith, Sarah K.
    Chiang, Chen-Yuan
    Conradie, Francesca
    Dalai, Doljinsuren
    van Deun, Armand
    Phan-Thuong Dat
    Ngoc Lan
    Master, Iqbal
    Mebrahtu, Tesfamarium
    Meressa, Daniel
    Moodliar, Ronelle
    Ngubane, Nosipho
    Sanders, Karen
    Squire, Stephen Bertel
    Torrea, Gabriela
    Tsogt, Bazarragchaa
    Rusen, I. D.
    [J]. NEW ENGLAND JOURNAL OF MEDICINE, 2019, 380 (13) : 1201 - 1213