Literature DB >> 17971533

Statistical methods for analyzing sequentially randomized trials.

Oliver Bembom1, Mark J van der Laan.   

Abstract

In this issue of the Journal, Thall et al. present the results of a clinical trial that makes use of sequential randomization, a novel trial design that allows the investigator to study adaptive treatment strategies. Our aim is to complement this groundbreaking work by reviewing the current state of the art of statistical methods available for such analyses. Using the data collected by Thall et al. as an example, we focus on two different approaches for estimating the success rates of different adaptive treatment strategies of interest. By emphasizing the intuitive appeal and straightforward implementation of these methods and illustrating the striking findings to which these methods can lead, we hope to convince the reader that this novel trial design provides a rich source of information that is made readily accessible through current analytical approaches.

Mesh:

Year:  2007        PMID: 17971533      PMCID: PMC2610531          DOI: 10.1093/jnci/djm185

Source DB:  PubMed          Journal:  J Natl Cancer Inst        ISSN: 0027-8874            Impact factor:   13.506


  9 in total

1.  An experimental design for the development of adaptive treatment strategies.

Authors:  S A Murphy
Journal:  Stat Med       Date:  2005-05-30       Impact factor: 2.373

2.  Dynamic treatment regimes: practical design considerations.

Authors:  Philip W Lavori; Ree Dawson
Journal:  Clin Trials       Date:  2004-02       Impact factor: 2.486

3.  Effectiveness of atypical antipsychotic drugs in patients with Alzheimer's disease.

Authors:  Lon S Schneider; Pierre N Tariot; Karen S Dagerman; Sonia M Davis; John K Hsiao; M Saleem Ismail; Barry D Lebowitz; Constantine G Lyketsos; J Michael Ryan; T Scott Stroup; David L Sultzer; Daniel Weintraub; Jeffrey A Lieberman
Journal:  N Engl J Med       Date:  2006-10-12       Impact factor: 91.245

4.  Causal effect models for realistic individualized treatment and intention to treat rules.

Authors:  Mark J van der Laan; Maya L Petersen
Journal:  Int J Biostat       Date:  2007       Impact factor: 0.968

5.  Marginal Mean Models for Dynamic Regimes.

Authors:  S A Murphy; M J van der Laan; J M Robins
Journal:  J Am Stat Assoc       Date:  2001-12-01       Impact factor: 5.033

6.  Evaluating multiple treatment courses in clinical trials.

Authors:  P F Thall; R E Millikan; H G Sung
Journal:  Stat Med       Date:  2000-04-30       Impact factor: 2.373

7.  Effects of antipsychotic medications on psychosocial functioning in patients with chronic schizophrenia: findings from the NIMH CATIE study.

Authors:  Marvin S Swartz; Diana O Perkins; T Scott Stroup; Sonia M Davis; George Capuano; Robert A Rosenheck; Fred Reimherr; Mark F McGee; Richard S E Keefe; Joseph P McEvoy; John K Hsiao; Jeffrey A Lieberman
Journal:  Am J Psychiatry       Date:  2007-03       Impact factor: 18.112

8.  Adaptive therapy for androgen-independent prostate cancer: a randomized selection trial of four regimens.

Authors:  Peter F Thall; Christopher Logothetis; Lance C Pagliaro; Sijin Wen; Melissa A Brown; Dallas Williams; Randall E Millikan
Journal:  J Natl Cancer Inst       Date:  2007-10-30       Impact factor: 13.506

9.  Depression, IV: STAR*D treatment trial for depression.

Authors:  A John Rush; Madhukar Trivedi; Maurizio Fava
Journal:  Am J Psychiatry       Date:  2003-02       Impact factor: 18.112

  9 in total
  12 in total

1.  Collaborative double robust targeted maximum likelihood estimation.

Authors:  Mark J van der Laan; Susan Gruber
Journal:  Int J Biostat       Date:  2010-05-17       Impact factor: 0.968

2.  Targeted maximum likelihood based causal inference: Part II.

Authors:  Mark J van der Laan
Journal:  Int J Biostat       Date:  2010-02-22       Impact factor: 0.968

3.  Innovative Clinical Trial Designs: Toward a 21st-Century Health Care System.

Authors:  Tze L Lai; Philip W Lavori
Journal:  Stat Biosci       Date:  2011-12

4.  Comparing cluster-level dynamic treatment regimens using sequential, multiple assignment, randomized trials: Regression estimation and sample size considerations.

Authors:  Timothy NeCamp; Amy Kilbourne; Daniel Almirall
Journal:  Stat Methods Med Res       Date:  2017-06-19       Impact factor: 3.021

5.  Improving Patient Reported Outcomes and Preventing Depression and Anxiety in Older Adults With Knee Osteoarthritis: Results of a Sequenced Multiple Assignment Randomized Trial (SMART) Study.

Authors:  Jordan F Karp; Jun Zhang; Abdus S Wahed; Stewart Anderson; Mary Amanda Dew; G Kelley Fitzgerald; Debra K Weiner; Steve Albert; Ari Gildengers; Meryl Butters; Charles F Reynolds
Journal:  Am J Geriatr Psychiatry       Date:  2019-03-21       Impact factor: 4.105

Review 6.  Innovative designs of point-of-care comparative effectiveness trials.

Authors:  Mei-Chiung Shih; Mintu Turakhia; Tze Leung Lai
Journal:  Contemp Clin Trials       Date:  2015-06-19       Impact factor: 2.226

7.  Discussion of Evaluation of Viable Dynamic Treatment Regimes in a Sequentially Randomized Trial of Advanced Prostate Cancer, by Wang et al. 2012.

Authors:  Paul Chaffee; Mark van der Laan
Journal:  J Am Stat Assoc       Date:  2012-07-24       Impact factor: 5.033

8.  Targeted maximum likelihood estimation for dynamic treatment regimes in sequentially randomized controlled trials.

Authors:  Paul H Chaffee; Mark J van der Laan
Journal:  Int J Biostat       Date:  2012-06-22       Impact factor: 0.968

9.  Challenges and Solutions for Depression Prevention Research: Methodology for a Depression Prevention Trial for Older Adults with Knee Arthritis and Emotional Distress.

Authors:  Jordan F Karp; Mary Amanda Dew; Abdus S Wahed; Kelley Fitzgerald; Chloe A Bolon; Debra K Weiner; Jennifer Q Morse; Steve Albert; Meryl Butters; Ariel Gildengers; Charles F Reynolds
Journal:  Am J Geriatr Psychiatry       Date:  2015-11-17       Impact factor: 4.105

10.  SMART designs in cancer research: Past, present, and future.

Authors:  Kelley M Kidwell
Journal:  Clin Trials       Date:  2014-04-14       Impact factor: 2.486

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