Noninferiority testing with censoring when the event rate is low.
Kaplan-Meier estimates
Tuberculosis
exact tests
loss to follow-up
noninferiority
Journal
Statistics in medicine
ISSN: 1097-0258
Titre abrégé: Stat Med
Pays: England
ID NLM: 8215016
Informations de publication
Date de publication:
10 11 2022
10 11 2022
Historique:
revised:
19
07
2022
received:
07
04
2021
accepted:
29
07
2022
pubmed:
23
8
2022
medline:
22
10
2022
entrez:
22
8
2022
Statut:
ppublish
Résumé
The PREDICT TB trial tests noninferiority of an abbreviated treatment regimen (arm A) vs a conventional treatment regimen (arm C). Treatment trials of drug-susceptible tuberculosis are expected to have low event rates (ie, relapse probabilities around 3-5%). We examine the question of what is the "best" way to test for noninferiority in a setting with low event rates. In a series of simulations supported by theoretical arguments, we examine operating characteristics of five tests, including normal approximation, exact, and simulation-based tests. Two of these tests are constructed from Kaplan-Meier based-estimators, which account for variable follow-up time (and those lost to follow-up). We evaluate the effect of loss to follow-up via simulations. We also examine the results of the five tests on a data set similar to PREDICT TB, the REMoxTB trial. We find that the normal approximation tests perform well, albeit with small type I error rate inflation. We also find that the Kaplan-Meier methods generally have larger power than the other tests, especially when there is between 10-30% loss to follow-up.
Types de publication
Journal Article
Research Support, N.I.H., Extramural
Langues
eng
Sous-ensembles de citation
IM
Pagination
5102-5112Informations de copyright
© 2022 John Wiley & Sons Ltd.
Références
Johnson JL, Hadad DJ, Dietze R, et al. Shortening treatment in adults with Noncavitary tuberculosis and 2-month culture conversion. Am J Respir Crit Care Med. 2009;180(6):558-563.
Gillespie SH, Crook AM, McHugh TD, et al. Four-month Moxifloxacin-based regimens for drug-sensitive tuberculosis. New Engl J Med. 2014;371(17):1577-1587.
Jindani A, Harrison TS, Nunn AJ, et al. High-dose Rifapentine with Moxifloxacin for pulmonary tuberculosis. N Engl J Med. 2014;371:1599-1608.
Merle CS, Fielding K, Sow OB, et al. A four-month Gatifloxacin-containing regimen for treating tuberculosis. New Engl J Med. 2014;371(17):1588-1598.
Parzen E. Modern Probability Theory and its Applications. New York: John Wiley & Sons (Wiley Classics Library Edition); 1992.
Proschan MA, Shaw PA. Essentials of Probability Theory for Statisticians. CRC Press: Chapman & Hall; 2016.
Crans GG, Shuster JJ. How conservative is Fisher's exact test? A quantitative evaluation of the two-sample comparative binomial trial. Stat Med. 2008;27(18):3598-3611.
Fay MP, Brittain EH, Proschan MA. Pointwise confidence intervals for a survival distribution with small samples or heavy censoring. Biostatistics. 2013;14(4):723-736.
Hall P. Theoretical comparison of bootstrap confidence intervals. Ann Stat. 1988;16(3):927-953.
Barnard GA. A new test for 2×$$ \times $$ 2 tables. Nature. 1945;156:177.
Boschloo RD. Raised conditional level of significance for the 2 × 2-table when testing the equality of two probabilities. Stat Neerl, 1970;24(1):1-9.
Fay MP, Proschan MA, Brittain E. Combining one-sample confidence procedures for inference in the two-sample case. Biometrics. 2015;71(1):146-156.
Agresti A, Coull BA. Approximate is better than “exact” for interval estimation of binomial proportions. Am Stat. 1998;52(2):119-126.
Food and Drug Administration. Application number: 21-024/S-005. Accessed January, 2022. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2000/21024s5_Priftin_statr.pdf. 2000.