An experimental design for the development of adaptive treatment strategies
Abstract
In adaptive treatment strategies, the treatment level and type is repeatedly adjusted according to ongoing individual response. Since past treatment may have delayed effects, the development of these treatment strategies is challenging. This paper advocates the use of sequential multiple assignment randomized trials in the development of adaptive treatment strategies. Both a simple ad hoc method for ascertaining sample sizes and simple analysis methods are provided. Copyright © 2004 John Wiley & Sons, Ltd.
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
SMART designs make no a priori assumptions about the existence or form of delayed treatment effects.
“SMART designs make no a priori assumptions about the existence or form of delayed treatment effects [51, 52]”
From A Sequential Multiple Assignment Randomized Trial (SMART) study of medication and CBT sequencing in the treatment of pediatric anxiety disorders (2021) · cites this paper as [51]
SMART designs are ideal for developing an adaptive intervention that optimizes outcomes based on patient treatment history.
“making them ideal for developing an adaptive intervention that optimizes outcomes based on patient treatment history [51–54].”
From A Sequential Multiple Assignment Randomized Trial (SMART) study of medication and CBT sequencing in the treatment of pediatric anxiety disorders (2021) · cites this paper as [51]
This will be a single-blind SMART design of 24-week treatment duration that will employ two sequential levels of randomization, one in each of two 12-week stages.
“This will be a single-blind [126, 127] SMART design [51, 52] of 24-week treatment duration that will employ two sequential levels of randomization, one in each of two 12-week stages of the study (Fig. 2).”
From A Sequential Multiple Assignment Randomized Trial (SMART) study of medication and CBT sequencing in the treatment of pediatric anxiety disorders (2021) · cites this paper as [51]
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Cites
Reinforcement Learning: An Introduction
Sutton, R. S., & Barto, A. G. (1998) · IEEE Transactions on Neural Networks · doi:10.1109/tnn.1998.712192 · Cited by 27,225 · see citing works
Bellman, R. E. (2010) · doi:10.1515/9781400835386 · Cited by 14,588 · see citing works
Robins, J. (1986) · Mathematical Modelling · doi:10.1016/0270-0255(86)90088-6 · Cited by 2,451 · see citing works
Flagged first, then by citations
Research works printed but not linked: 38
- Glasgow MS, Engel BT, D'Lugo BC. A controlled study of a standardized behavioural stepped treatment for hypertension. Psychosomatic Medicine 1989; 51:10 -26.
- Kreuter MW, Strecher VJ. Do tailored behavior change messages enhance the e ectiveness of health risk appraisal? Results from a randomized trial. Health Education Research 1996; 11(1):97-105.
- Breslin FC, Sobell MB, Sobell LC, Cunningham JA, Sdao-Jarvie K, Borsoi D. Problem drinkers: evaluation of a stepped-care approach. Journal of Substance Abuse 1999; 10(3):217-232.
- Un tzer J, Katon W, Williams JW, Callahan CM, Harpole L, Hunkeler EM, Ho ng M, Arean P, Hegel MT, Schoenbaum M, Oishi SM, Langston CA. Improving primary care for depression in late life: the design of a multicenter randomized trial. Medical Care 2001; 39(8):785-799.
- Brooner RK, Kidorf M. Using behavioral reinforcement to improve methadone treatment participation. Science and Practice Perspectives 2002; 1:38-48.
- Lavori PW, Dawson R, Roth AJ. Flexible treatment strategies in chronic disease: clinical and research implications. Biological Psychiatry 2000; 48:605-614.
- Copyright ? 2004 John Wiley & Sons, Ltd. Statist. Med. 2005; 24:1455-1481
- Robins JM. Information recovery and bias adjustment in proportional hazards regression analysis of randomized trials using surrogate markers. Proceedings of the Biopharmaceutical Section; American Statistical Association 1993; 24-33.
- Robins JM. Causal inference from complex longitudinal data. Latent Variable Modeling and Applications to Causality, Berkane M (ed.). Lecture Notes in Statistics, vol. 120. Springer: New York, 1997; 69-117.
- Murphy SA, van der Laan MJ, Robins JM, CPPRG. Marginal mean models for dynamic regimes. Journal of American Statistical Association 2001; 96:1410 -1423.
- Lavori PW, Dawson R. Dynamic treatment regimes: practical design considerations. Clinical Trials 2003; 1: 9-20.
- Schneider LS, Tariot PN, Lyketsos CG, Dagerman KS, Davis KL, Davis S, Hsiao JK, Jeste DV, Katz IR, Olin JT, Pollock BG, Rabins PV, Rosenheck RA, Small GW, Lebowitz B, Lieberman JA. National Institute of Mental Health clinical antipsychotic trials of intervention e ectiveness (CATIE), Alzheimer disease trial methodology. American Journal of Geriatric Psychiatry 2001; 9(4):346-360.
- Rush AJ, Trivedi M, Fava M. Depression, IV. American Journal of Psychiatry 2003; 160(2):237.
- Lavori PW, Rush AJ, Wisniewski SR, Alpert J, Fava M, Kupfer DJ, Nierenberg A, Quitkin FM, Sacheim HA, Thase ME, Trivedi M. Biological Psychiatry 2001; 50:792-801.
- Thall PF, Millikan RE, Sung HG. Evaluating multiple treatment courses in clinical trials. Statistics and Medicine 2000; 19:1011-1028.
- Berry DA. Adaptive trials and Bayesian statistics in drug development. Biopharmaceutical Report, vol. 9, 2001; 1-7.
- Rosenberger WF, Stallard N, Ivanova A, Harper CN, Ricks ML. Optimal adaptive designs for binary response trials. Biometrics 2001; 57:909-913.
- Fisher LD. Self-designing clinical trials. Statistics in Medicine 1998; 17:1551-1562.
- Jennison C, Turnbull BW. Mid-course sample size modiÿcation in clinical trials based on the observed treatment e ect. Statistics in Medicine 2003; 22:971-993.
- Cowell RG, Dawid AP, Lauritzen SL, Spiegelhalter DJ. Probabilistic Networks and Expert Systems. Springer: New York, 1999.
- Gail MH, Benichou J (eds). Encyclopedia of Epidemiologic Methods. Wiley: Chichester, New York, 2000.
- Parmigiani G. Modeling in Medical Decision Making: A Bayesian Approach. Wiley: England, 2002.
- Tengs TO, Winer EP, Paddock S, Aguilar-Chavez, Berry DA. Testing for the BRCA1 and BRCA2 breast-ovarian cancer susceptibility genes: a decision analysis. Medical Decision Making 1998; 18:365-375.
- Lavori PW, Dawson R. A design for testing clinical strategies: biased individually tailored within-subject randomization. Journal of the Royal Statistical Society A 2000; 163:29 -38.
- Box GP, Hunter WG, Hunter JS. Statistics for Experimenters. Wiley: New York, 1978.
- Robins JM. Robust estimation in sequentially ignorable missing data models and causal inference models. Proceedings of the American Statistical Association. Section on Bayesian Statistical Science 1999; 6-10.
- Murphy SA. Optimal dynamic treatment regimes. Journal of the Royal Statistical Society; Series B 2003; 65(2):331-366.
- Berry DA, Eick SG. Adaptive assignment versus balanced randomization in clinical trials: a decision analysis. Statistics in Medicine 1995; 14:231-246.
- Berry DA. General keynote: clinical trial design. Gynecologic Oncology 2003; 88:S114-S116.
- Thall PF, Sung HG, Estey EH. Selecting therapeutic strategies based on e cacy and death in multicourse clinical trials. Journal of the American Statistical Association 2002; 97:29-39.
- Rubin DB. Bayesian inference for causal e ects: the role of randomization. The Annals of Statistics 1978; 6:34-58.
- Rubin DB. Comment: which ifs have causal answers. Journal of the American Statistical Association 1986; 81:961-962.
- Copyright ? 2004 John Wiley & Sons, Ltd. Statist. Med. 2005; 24:1455-1481
- Robins JM. Addendum to a new approach to causal inference in mortality studies with sustained exposure periods-application to control of the healthy worker survivor e ect. Computers and Mathematics with Applications 1987; 14:923-945.
- Ser ing RJ. Approximation theorems of Mathematical Statistics. Wiley: New York, 1980.
- Prochaska JO, Velicer WF, Guadagnoli E, Rossi JS, DiClemente CC. Patterns of change: dynamic typology applied to smoking cessation. Multivariate Behavioral Research 1991; 26(1):83-107.
- Prochaska JO, Velicer WF, Fava JL, Rossi JS, Tsoh JY. Evaluating a population-based recruitment approach and a stage-based expert system intervention for smoking cessation. Addictive Behaviors 2001; 26:583-602.
- Copyright ? 2004 John Wiley & Sons, Ltd. Statist. Med. 2005; 24:1455-1481
Cited by
Sorted by citations
Collins, L. M., Murphy, S. A., & Strecher, V. (2007) · American Journal of Preventive Medicine · doi:10.1016/j.amepre.2007.01.022 · Cited by 1,082 · see citing works
Self-citationEvidence-based psychosocial treatments for attention-deficit/hyperactivity disorder
Pelham, W. E., & Fabiano, G. A. (2008) · Journal of Clinical Child & Adolescent Psychology · doi:10.1080/15374410701818681 · Cited by 917 · see citing works
A "SMART" Design for Building Individualized Treatment Sequences
Lei, H., Nahum-Shani, I., Lynch, K., Oslin, D., & Murphy, S. A. (2012) · Annual Review of Clinical Psychology · doi:10.1146/annurev-clinpsy-032511-143152 · Cited by 557 · see citing works
Self-citationmHealth: A Mechanism to Deliver More Accessible, More Effective Mental Health Care
Price, M., Yuen, E. K., Goetter, E. M., Herbert, J. D., Forman, E. M., Acierno, R., & Ruggiero, K. J. (2014) · Clinical Psychology & Psychotherapy · doi:10.1002/cpp.1855 · Cited by 488 · see citing works
Almirall, D., Nahum-Shani, I., Sherwood, N. E., & Murphy, S. A. (2014) · Translational Behavioral Medicine · doi:10.1007/s13142-014-0265-0 · Cited by 453 · see citing works
Self-citationDrake, R. E., O'Neal, E. L., & Wallach, M. A. (2008) · Journal of Substance Abuse Treatment · doi:10.1016/j.jsat.2007.01.011 · Cited by 447 · see citing works
Reinforcement learning for intelligent healthcare applications: A survey
Coronato, A., Naeem, M., De Pietro, G., & Paragliola, G. (2020) · Artificial Intelligence in Medicine · doi:10.1016/j.artmed.2020.101964 · Cited by 320 · see citing works
Experimental design and primary data analysis methods for comparing adaptive interventions.
Nahum-Shani, I., Qian, M., Almirall, D., Pelham, W. E., Gnagy, B., Fabiano, G. A., Waxmonsky, J. G., Yu, J., & Murphy, S. A. (2012) · Psychological Methods · doi:10.1037/a0029372 · Cited by 298 · see citing works
Self-citationPelham, W. E., Fabiano, G. A., Waxmonsky, J. G., Greiner, A. R., Gnagy, E. M., Pelham, W. E., Coxe, S., Verley, J., Bhatia, I., Hart, K., et al. (2016) · Journal of Clinical Child & Adolescent Psychology · doi:10.1080/15374416.2015.1105138 · Cited by 251 · see citing works
Self-citationAlmirall, D., Compton, S. N., Gunlicks‐Stoessel, M., Duan, N., & Murphy, S. A. (2012) · Statistics in Medicine · doi:10.1002/sim.4512 · Cited by 223 · see citing works
Self-citationAdvancing the science of mHealth
Nilsen, W., Kumar, S., Shar, A., Varoquiers, C., Wiley, T., Riley, W. T., Pavel, M., & Atienza, A. A. (2012) · Journal of Health Communication · doi:10.1080/10810730.2012.677394 · Cited by 226 · see citing works
Adaptive Designs for Randomized Trials in Public Health
Brown, C. H., Ten Have, T. R., Jo, B., Dagne, G., Wyman, P. A., Muthén, B., & Gibbons, R. D. (2009) · Annual Review of Public Health · doi:10.1146/annurev.publhealth.031308.100223 · Cited by 184 · see citing works
Rivera, D. E., Pew, M. D., & Collins, L. M. (2007) · Drug and Alcohol Dependence · doi:10.1016/j.drugalcdep.2006.10.020 · Cited by 151 · see citing works
Evaluating the Impact of Physical Activity Apps and Wearables: Interdisciplinary Review
McCallum, C., Rooksby, J., & Gray, C. M. (2018) · JMIR mhealth and uhealth · doi:10.2196/mhealth.9054 · Cited by 126 · see citing works
Adaptive Treatment Strategies in Chronic Disease
Lavori, P. W., & Dawson, R. (2008) · Annual Review of Medicine · doi:10.1146/annurev.med.59.062606.122232 · Cited by 113 · see citing works
Wang, L., Rotnitzky, A., Lin, X., Millikan, R. E., & Thall, P. F. (2012) · Journal of the American Statistical Association · doi:10.1080/01621459.2011.641416 · Cited by 108 · see citing works
“A number of recent articles discuss design of randomized trials that aim at evaluating DTRs rather than individual treatments (Lavori and Dawson 2000, 2004; Thall, Millikan, and Sung 2000; Thall, Sung, and Estey 2002; Murphy 2005; Oetting et al. 2011).”
BackgroundInfluentialVlaeyen, J. W. S., Morley, S., & Crombez, G. (2016) · Behaviour Research and Therapy · doi:10.1016/j.brat.2016.08.016 · Cited by 117 · see citing works
Treating Mental Health and Substance Use Disorders in Adolescents: What Is on the Menu?
Brewer, S., Godley, M. D., & Hulvershorn, L. A. (2017) · Current Psychiatry Reports · doi:10.1007/s11920-017-0755-0 · Cited by 79 · see citing works
Dahne, J., Lejuez, C. W., Diaz, V. A., Player, M. S., Kustanowitz, J., Felton, J. W., & Carpenter, M. J. (2019) · Behavior Therapy · doi:10.1016/j.beth.2018.12.003 · Cited by 78 · see citing works
Treatment approaches for functional neurological disorders in children
Vassilopoulos, A., Mohammad, S., Dure, L., Kozlowska, K., & Fobian, A. D. (2022) · Current Treatment Options in Neurology · doi:10.1007/s11940-022-00708-5 · Cited by 70 · see citing works
Cheung, Y. K., Chakraborty, B., & Davidson, K. W. (2015) · Biometrics · doi:10.1111/biom.12258 · Cited by 55 · see citing works
Stepped care in the treatment of trichotillomania
Rogers, K., Banis, M., Falkenstein, M. J., Malloy, E. J., McDonough, L., Nelson, S. O., Rusch, N., & Haaga, D. A. F. (2014) · Journal of Consulting and Clinical Psychology · doi:10.1037/a0035744 · Cited by 59 · see citing works
The future of psychological treatments: The Marburg Declaration
Rief, W., Asmundson, G. J. G., Bryant, R. A., Clark, D. M., Ehlers, A., Holmes, E. A., McNally, R. J., Neufeld, C. B., Wilhelm, S., Jaroszewski, A. C., et al. (2024) · Clinical Psychology Review · doi:10.1016/j.cpr.2024.102417 · Cited by 54 · see citing works
Czyz, E. K., King, C. A., Prouty, D., Micol, V. J., Walton, M., & Nahum‐Shani, I. (2021) · Journal of Child Psychology and Psychiatry · doi:10.1111/jcpp.13383 · Cited by 54 · see citing works
A decision support framework to implement optimal personalized marketing interventions
Guelman, L., Guillén, M., & Pérez-Marín, A. M. (2015) · Decision Support Systems · doi:10.1016/j.dss.2015.01.010 · Cited by 48 · see citing works
McGorry, P. D., Mei, C., Amminger, G. P., Yuen, H. P., Kerr, M., Spark, J., Wallis, N., Polari, A., Baird, S., Buccilli, K., et al. (2023) · JAMA Psychiatry · doi:10.1001/jamapsychiatry.2023.1947 · Cited by 48 · see citing works
Erickson, M. L., Allen, J. M., Beavers, D. P., Collins, L. M., Davidson, K. W., Erickson, K. I., Esser, K. A., Hesselink, M. K. C., Moreau, K. L., Laber, E. B., et al. (2023) · GeroScience · doi:10.1007/s11357-022-00668-3 · Cited by 47 · see citing works
SMARTer Approach to Personalizing Intervention for Children With Autism Spectrum Disorder
Kasari, C., Sturm, A., & Shih, W. (2018) · Journal of Speech, Language, and Hearing Research · doi:10.1044/2018_jslhr-l-rsaut-18-0029 · Cited by 46 · see citing works
Cillessen, L., van de Ven, M. O., Compen, F. R., Bisseling, E. M., van der Lee, M. L., & Speckens, A. E. (2020) · Journal of Medical Internet Research · doi:10.2196/17526 · Cited by 43 · see citing works
Screening Experiments for Developing Dynamic Treatment Regimes
Murphy, S. A., & Bingham, D. (2009) · Journal of the American Statistical Association · doi:10.1198/jasa.2009.0119 · Cited by 41 · see citing works
Self-citationRamchand, R., Griffin, B. A., Slaughter, M. E., Almirall, D., & McCaffrey, D. F. (2014) · Journal of Substance Abuse Treatment · doi:10.1016/j.jsat.2014.06.012 · Cited by 18 · see citing works
Smith, S. K., Somers, T. J., Kuhn, E., Laber, E., Sung, A. D., Syrjala, K. L., Feger, B., Kelleher, S. A., Majestic, C., Gebert, R., et al. (2021) · Contemporary Clinical Trials · doi:10.1016/j.cct.2021.106569 · Cited by 14 · see citing works
Newcomb, M. E., Swann, G., Mohr, D., & Mustanski, B. (2018) · AIDS and Behavior · doi:10.1007/s10461-018-2027-3 · Cited by 12 · see citing works
Peterson, B. S., West, A. E., Weisz, J. R., Mack, W. J., Kipke, M. D., Findling, R. L., Mittman, B. S., Bansal, R., Piantadosi, S., Takata, G., et al. (2021) · BMC Psychiatry · doi:10.1186/s12888-021-03314-y · Cited by 13 · see citing works
Buyze, J., Van Rompaye, B., & Goetghebeur, E. (2010) · Statistics in Medicine · doi:10.1002/sim.3856 · Cited by 9 · see citing works
Badger, T., Segrin, C., Crane, T. E., Chalasani, P., Arslan, W., Hadeed, M., Cunicelli, N., Given, C. W., & Sikorskii, A. (2026) · Journal of Cancer Survivorship · doi:10.1007/s11764-024-01723-y · Cited by 1 · see citing works
- Authors
- Murphy, S. A.
- Venue
- Statistics in Medicine
- Publisher
- Wiley
- Published
- 2004-12-07
- Volume
- 24
- Issue
- 10
- Pages
- 1455-1481
- ISSN
- 0277-6715
- 1097-0258
- Cited by
- 595
- References
- 3
- Language
- en
- Licence
- Funders
- NIDA (K02-DA 15672, P50 DA 10075)
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review