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@ARTICLE{Edelmann:147214,
author = {D. Edelmann$^*$ and C. Habermehl and R. F. Schlenk and A.
Benner$^*$},
title = {{A}djusting {S}imon's optimal two-stage design for
heterogeneous populations based on stratification or using
historical controls.},
journal = {Biometrical journal},
volume = {62},
number = {2},
issn = {1521-4036},
address = {Berlin},
publisher = {Wiley-VCH},
reportid = {DKFZ-2019-02340},
pages = {311-329},
year = {2020},
note = {2020 Mar;62(2):311-329#EA:C060#LA:C060#},
abstract = {In many cancer studies, the population under consideration
is highly heterogeneous in terms of clinical, demographical,
and biological covariates. As the covariates substantially
impact the individual prognosis, the response probabilities
of patients entering the study may strongly vary. In this
case, the operating characteristics of classical clinical
trial designs heavily depend on the covariates of patients
entering the study. Notably, both type I and type II errors
can be much higher than specified. In this paper, two
modifications of Simon's optimal two-stage design correcting
for heterogeneous populations are derived. The first
modification assumes that the patient population is divided
into a finite number of subgroups, where each subgroup has a
different response probability. The second approach uses a
logistic regression model based on historical controls to
estimate the response probabilities of patients entering the
study. The performance of both approaches is demonstrated
using simulation examples.},
cin = {C060},
ddc = {570},
cid = {I:(DE-He78)C060-20160331},
pnm = {313 - Cancer risk factors and prevention (POF3-313)},
pid = {G:(DE-HGF)POF3-313},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:31553076},
doi = {10.1002/bimj.201800390},
url = {https://inrepo02.dkfz.de/record/147214},
}