Using short-term response information to facilitate adaptive randomization for survival clinical trials.

Full Abstract

Increased survival is a common goal of cancer clinical trials. Owing to the long periods of observation and follow-up to assess patient survival outcome, it is difficult to use outcome-adaptive randomization in these trials. In practice, often information about a short-term response is quickly available during or shortly after treatment, and this short-term response is a good predictor for long-term survival. For example, complete remission of leukemia can be achieved and measured after a few cycles of treatment. It is a short-term response that is desirable for prolonging survival. We propose a new design for survival trials when such short-term response information is available. We use the short-term information to 'speed up' the adaptation of the randomization procedure. We establish a connection between the short-term response and the long-term survival through a Bayesian model, first by using prior clinical information, and then by dynamically updating the model according to information accumulated in the ongoing trial. Interim monitoring and final decision making are based upon inference on the primary outcome of survival. The new design uses fewer patients, and can more effectively assign patients to the better treatment arms. We demonstrate these properties through simulation studies. (c) 2009 John Wiley & Sons, Ltd.

Author information

Author/s: Huang, Xuelin (X); Ning, Jing (J); Li, Yisheng (Y); Estey, Elihu (E); Issa, Jean-Pierre (JP); Berry, Donald A (DA);

Affiliation: Department of Biostatistics, The University of Texas, M. D. Anderson Cancer Center, Houston, TX 77030, U.S.A.

Grants: 1 P50 CA100632 (Agency:NCI NIH HHS) ; 1 PO1 CA108631-01 (Agency:NCI NIH HHS)

Journal and publication information

Publication Type: Journal Article; Research Support, N.I.H., Extramural

Journal: Statistics in medicine (Stat Med), published in England. (Language: eng)

Reference: 2009-May; vol 28 (issue 12) : pp 1680-9

Dates: Created 2009/05/07; Completed 2009/07/01;

PMID: 19326367, status: MEDLINE (last retrieval date: 7/1/2009, IMS Date: )

Sourced from the National Library of Medicine. Abstract text and other information may be subject to copyright.

External Links for this article
(including full text providers, if available):

Click Electronic Full-text Provider Links to see options for finding the electronic full text links to this article. Note there may be a subscription or fee required for access to the full text. See our FAQ for information on finding FREE full text articles.

This article may also be located in paper journal collections available in many libraries. Use the Journal and Publication Information above to find the full article.

MeSH headings (categories)

This article was linked to the MESH Headings shown below.

This article has not been indexed for related articles as yet, however you can still use the live related article search links below.

See 100+ related articles.

See a large map of 100+ related articles.