Universität zu Köln

Danzer, Moritz Fabian ORCID: 0000-0002-0512-4227, Terzer, Tobias, Berthold, Frank, Faldum, Andreas and Schmidt, Rene (2022). Confirmatory adaptive group sequential designs for single-arm phase II studies with multiple time-to-event endpoints. Biom. J., 64 (2). S. 312 - 343. HOBOKEN: WILEY. ISSN 1521-4036

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Abstract

Existing methods concerning the assessment of long-term survival outcomes in one-armed trials are commonly restricted to one primary endpoint. Corresponding adaptive designs suffer from limitations regarding the use of information from other endpoints in interim design changes. Here we provide adaptive group sequential one-sample tests for testing hypotheses on the multivariate survival distribution derived from multi-state models, while making provision for data-dependent design modifications based on all involved time-to-event endpoints. We explicitly elaborate application of the methodology to one-sample tests for the joint distribution of (i) progression-free survival (PFS) and overall survival (OS) in the context of an illness-death model, and (ii) time to toxicity and time to progression while accounting for death as a competing event. Large sample distributions are derived using a counting process approach. Small sample properties are studied by simulation. An already established multi-state model for non-small cell lung cancer is used to illustrate the adaptive procedure.

Item Type:	Journal Article
Creators:	Creators Email ORCID ORCID Put Code Danzer, Moritz Fabian UNSPECIFIED orcid.org/0000-0002-0512-4227 UNSPECIFIED Terzer, Tobias UNSPECIFIED UNSPECIFIED UNSPECIFIED Berthold, Frank UNSPECIFIED UNSPECIFIED UNSPECIFIED Faldum, Andreas UNSPECIFIED UNSPECIFIED UNSPECIFIED Schmidt, Rene UNSPECIFIED UNSPECIFIED UNSPECIFIED
URN:	urn:nbn:de:hbz:38-583508
DOI:	10.1002/bimj.202000205
Journal or Publication Title:	Biom. J.
Volume:	64
Number:	2
Page Range:	S. 312 - 343
Date:	2022
Publisher:	WILEY
Place of Publication:	HOBOKEN
ISSN:	1521-4036
Language:	English
Faculty:	Unspecified
Divisions:	Unspecified
Subjects:	no entry
Uncontrolled Keywords:	Keywords Language INTERIM ANALYSES; CLINICAL-TRIALS; SURVIVAL-DATA; SAMPLE-SIZE; MODEL; SCHEDULE Multiple languages Mathematical & Computational Biology; Statistics & Probability Multiple languages
URI:	http://kups.ub.uni-koeln.de/id/eprint/58350