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2015 ; 24
(6
): 909-19
Nephropedia Template TP
gab.com Text
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English Wikipedia
The choice of test in phase II cancer trials assessing continuous tumour
shrinkage when complete responses are expected
#MMPMID22179821
Wason JM
; Mander AP
Stat Methods Med Res
2015[Dec]; 24
(6
): 909-19
PMID22179821
show ga
Traditionally, phase II cancer trials test a binary endpoint formed from a
dichotomisation of the continuous change in tumour size. Directly testing the
continuous endpoint provides considerable gains in power, although also results
in several statistical issues. One such issue is when complete responses, i.e.
complete tumour removal, are observed in multiple patients; this is a problem
when normality is assumed. Using simulated data and a recently published phase II
trial, we investigate how the choice of test affects the operating
characteristics of the trial. We propose using parametric tests based on the
censored normal distribution, comparing them to the t-test and Wilcoxon
non-parametric test. The censored normal distribution fits the real dataset well,
but simulations indicate its type-I error rate is inflated, and its power is only
slightly higher than the t-test. The Wilcoxon test has deflated type I error. For
two-arm designs, the differences are much smaller. We conclude that the t-test is
suitable for use when complete responses are present, although positively skewed
data can result in the non-parametric test having higher power.
|Antineoplastic Agents/*therapeutic use
[MESH]
|Clinical Trials, Phase I as Topic/*methods
[MESH]
|Data Interpretation, Statistical
[MESH]
|Endpoint Determination/*methods
[MESH]
|Humans
[MESH]
|Models, Statistical
[MESH]
|Neoplasms/*drug therapy/pathology
[MESH]
|Normal Distribution
[MESH]
|Randomized Controlled Trials as Topic/methods
[MESH]