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@ARTICLE{Labrenz:182282,
author = {J. Labrenz$^*$ and D. Edelmann$^*$ and J. Heitmann$^*$ and
H. Salih$^*$ and A. Kopp-Schneider$^*$ and R. Schlenk$^*$},
title = {{P}erformance of phase-{I} dose finding designs with and
without a run-in intra-patient dose escalation stage.},
journal = {Pharmaceutical statistics},
volume = {22},
number = {2},
issn = {1539-1604},
address = {New York, NY},
publisher = {Wiley},
reportid = {DKFZ-2022-02542},
pages = {236-247},
year = {2023},
note = {#LA:C060#LA:W010# / 2023 Mar;22(2):236-247},
abstract = {Dose-finding designs for phase-I trials aim to determine
the recommended phase-II dose (RP2D) for further phase-II
drug development. If the trial includes patients for whom
several lines of standard therapy failed or if the toxicity
of the investigated agent does not necessarily increase with
dose, optimal dose-finding designs should limit the
frequency of treatment with suboptimal doses. We propose a
two-stage design strategy with a run-in intra-patient dose
escalation part followed by a more traditional dose-finding
design. We conduct simulation studies to compare the 3 + 3
design, the Bayesian Optimal Interval Design (BOIN) and the
Continual Reassessment Method (CRM) with and without
intra-patient dose escalation. The endpoints are accuracy,
sample size, safety, and therapeutic efficiency. For
scenarios where the correct RP2D is the highest dose,
inclusion of an intra-patient dose escalation stage
generally increases accuracy and therapeutic efficiency.
However, for scenarios where the correct RP2D is below the
highest dose, intra-patient dose escalation designs lead to
increased risk of overdosing and an overestimation of RP2D.
The magnitude of the change in operating characteristics
after including an intra-patient stage is largest for the 3
+ 3 design, decreases for the BOIN and is smallest for the
CRM.},
keywords = {dose escalation (Other) / dose-finding (Other) /
intra-patient (Other) / maximum tolerated dose (Other) /
phase-I (Other)},
cin = {C060 / TU01},
ddc = {610},
cid = {I:(DE-He78)C060-20160331 / I:(DE-He78)TU01-20160331},
pnm = {313 - Krebsrisikofaktoren und Prävention (POF4-313)},
pid = {G:(DE-HGF)POF4-313},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:36285348},
doi = {10.1002/pst.2268},
url = {https://inrepo02.dkfz.de/record/182282},
}