A "SMART" design for building individualized treatment sequences

Annual Review of Clinical Psychology

Volumn 8, Issue , 2012, Pages 21-48

  Lei, H ^a   Nahum Shani, I ^a   Lynch, K ^b   Oslin, D ^b,c   Murphy, S A ^a,d  

^a UNIVERSITY OF MICHIGAN   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

^c VETERANS AFFAIRS MEDICAL CENTER   (United States)

^d UNIVERSITY OF MICHIGAN HEALTH SYSTEM   (United States)

Author keywords

adaptive intervention; individualized treatment sequences; Sequential Multiple Assignment Randomized Trial

Indexed keywords

METHYLPHENIDATE; NALTREXONE; PLACEBO;

ALCOHOL CONSUMPTION; ATTENTION DEFICIT DISORDER; BEHAVIOR MODIFICATION; COMPARATIVE STUDY; DATA ANALYSIS; DRUG ABUSE; EXPERIMENTAL DESIGN; HEALTH PROGRAM; HUMAN; INDIVIDUALIZATION; INTERVENTION STUDY; MEDICAL DECISION MAKING; PREGNANCY; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; SEQUENTIAL MULTIPLE ASSIGNMENT RANDOMIZED TRIAL; TREATMENT PLANNING; TREATMENT RESPONSE;

HUMANS; MENTAL DISORDERS; PATIENT-CENTERED CARE; PROGRAM DEVELOPMENT; PROGRAM EVALUATION; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RESEARCH DESIGN; UNITED STATES;

EID: 84861518319     PISSN: 15485943     EISSN: 15485951     Source Type: Book Series    
DOI: 10.1146/annurev-clinpsy-032511-143152     Document Type: Review

References (82)

1. Adamson LB, Bakeman R, Deckner DF. 2004. The development of symbol-infused joint engagement. Child Dev. 75:1171-87
2. Am. Acad. Child Adolesc. Psychiatry. 2007. Practice parameters for the assessment and treatment of children and adolescents with attention-deficit/ hyperactivity disorder. J. Am. Acad. Child Psychiatry 46:894-921
3. Anderson DK, Lord C, Risi S, Shulman C, Welch K, et al. 2007. Patterns of growth in verbal abilities among children with autism spectrum disorder. J. Consult. Clin. Psychol. 75:594-604
4. Angst J, Gamma A, Baldwin DS, Ajdacic-Gross V, Rossler W. 2009. The generalized anxiety spectrum: prevalence, onset, course and outcome. Eur. Arch. Psychiatry Clin. Neurosci. 259:37-45
5. Bandyopadhyay N, Dragalin V. 2007. Implementation of an adaptive group sequential design in a bioequivalence study. Pharmaceut. Statist. 6:115-22
6. Bierman KL, Nix RL, Maples JJ, Murphy SA. 2006. Examining clinical judgment in an adaptive intervention design: the Fast Track program. J. Consult. Clin. Psychol. 74:468-81
7. Brown RT, Antonuccio DO, DuPaul GJ, FristadMA, King CA, et al. 2007. Childhood Mental Health Disorders: Evidence Base and Contextual Factors for Psychosocial, Psychopharmacological, and Combined Interventions. Washington, DC: Am. Psychol. Assoc.
8. Cafiero J. 2005. Meaningful Exchanges for People with Autism: An Introduction to Augmentative and Alternative Communication. Bethesda, MD: Woodbine House
9. Chow SC, Chang M. 2008. Adaptive design methods in clinical trials-a review. Orphanet J. Rare Dis. 3:11
10. Ciraulo DA, Piechniczek-Buczek J, Iscan EN. 2003. Outcome predictors in substance use disorders. Psychiatr. Clin. North Am. 26:381-409
11. Collins LM, Murphy SA, Bierman KL. 2004. A conceptual framework for adaptive preventive interventions. Prev. Sci. 5:185-96
12. Crits-Christoph P, Siqueland L, Blaine J, Frank A, Luborsky L, et al. 1999. Psychosocial treatments for cocaine dependence-National Institute on Drug Abuse Collaborative Cocaine Treatment Study. Arch. Gen. Psychiatry 56:493-502
13. Dawson R, Lavori PW. 2011. Efficient design and inference formulti-stage randomized trials of individualized treatment policies. Biostatistics. In press
14. Dragalin V. 2006. Adaptive designs: terminology and classification. Drug. Inf. J. 40:425-35
15. Fabiano GA, Chacko A, Pelham WE, Robb J, Walker KS, et al. 2009. A comparison of behavioral parent training programs for fathers of children with attention-deficit/hyperactivity disorder. Behav. Ther. 40:190-204
16. Fabiano GA, Pelham WE, Waschbusch DA, Gnagy EM, Lahey BB, et al. 2006. A practical measure of impairment: psychometric properties of the impairment rating scale in samples of children with attention deficit hyperactivity disorder and two school-based samples. J. Clin. Child Adolesc. 35:369-85
17. Hammerness P, McCarthy K, Mancuso E, Gendron C, Geller D. 2009. Atomoxetine for the treatment of attention-deficit/hyperactivity disorder in children and adolescents: a review. Neuropsychiatr. Dis. Treat. 5:215-26
18. Hancock TB, Kaiser AP. 2006. Enhanced milieu teaching. In Treatment of Language Disorders in Children, ed. R McCauley, pp. 203-36. Baltimore, MD: Brookes
19. rnán MA, Brumback B, Robins JM. 2000. Marginal structural models to estimate the causal effect of zidovudine on the survival of HIV-positive men. Epidemiology 11:561-70
20. Hernán MA, Brumback B, Robins JM. 2002. Estimating the causal effect of zidovudine on CD4 count with a marginal structural model for repeated measures. Stat. Med. 21:1689-709
21. Honigmann P, Fischer H, Kurmann A, Audigé L, Schüpfer G, Metzger J. 2007. Investigating the effect of intra-operative infiltration with local anaesthesia on the development of chronic postoperative pain after inguinal hernia repair: a randomized placebo controlled triple blinded and group sequential study design [NCT00484731]. BMC Surg. 7:22
22. Hser YI, Longshore D, Anglin MD. 2007. The life course perspective on drug use: a conceptual framework for understanding drug use trajectories. Eval. Rev. 31:515-47
23. JonesH. 2010. Reinforcement-Based Treatment for Pregnant Drug Abusers (HOME II). Bethesda,MD: Natl. Inst. Health. http://clinicaltrials.gov/ct2/show/ NCT01177982?term=jones+pregnant&rank=9
24. Jones HE, O'Grady KE, Tuten M. 2011. Reinforcement-based treatment improves the maternal treatment and neonatal outcomes of pregnant patients enrolled in comprehensive care treatment. Am. J. Addict. 20:196-204
25. Jones HE, Wong CJ, Tuten M, Stitzer ML. 2005. Reinforcement-based therapy: 12-month evaluation of an outpatient drug-free treatment for heroin abusers. Drug Alcohol. Depend. 79:119-28
26. Joss RA, Alberto P, Bleher EA, Ludwig C, Siegenthaler P, et al. 1994. Combined-modality treatment of smallcell lung cancer: randomized comparison of three induction chemotherapies followed by maintenance chemotherapy with or without radiotherapy to the chest. Ann. Oncol. 5:921-28
27. Kasari C. 2009a. Developmental and Augmented Intervention for Facilitating Expressive Language (CCNIA). Bethesda, MD: Natl. Inst. Health. http://clinicaltrials.gov/ct2/show/NCT01013545?term= kasari&rank=5
28. Kasari C. 2009b. CCNIA Developmental and Augmented Intervention for Facilitating Expressive Language. Cent. Autism Res. Treat., Univ. Calif., Los Angeles. http://www.semel.ucla.edu/autism/research/ project/ccnia-developmental- augmented-intervention-facilitating-expressive-language
29. Kasari C, Freeman S, Paparella T. 2006. Joint attention and symbolic play in young children with autism: a randomized controlled intervention study. J. Child Psychol. Psychiatry 47:611-20
30. Kasari C, Paparella T, Freeman S, Jahromi LB. 2008. Language outcome in autism: randomized comparison of joint attention and play interventions. J. Consult. Clin. Psychol. 76:125-37
31. Lavori PW, Dawson R. 2000. A design for testing clinical strategies: biased adaptive within-subject randomization. J. R. Statist. Soc. A 163:29-38
32. Lavori PW, Dawson R. 2004. Dynamic treatment regimes: practical design considerations. Clin. Trials 1:9-20
33. Lavori PW, Dawson R, Rush AJ. 2000. Flexible treatment strategies in chronic disease: clinical and research implications. Biol. Psychiatry 48:605-14
34. Liu-Seifert H, Osuntokun OO, Feldman PD. 2012. Factors associated with adherence to treatment with olanzapine and other atypical antipsychoticmedications in patients with schizophrenia. Compr. Psychiatry. 53:107-15
35. Longabaugh R, Zweben A, Locastro JS, MillerWR. 2005. Origins, issues and options in the development of the combined behavioral intervention. J. Stud. Alcohol. 15(Suppl.):179-87
36. Lord C. 2000. Commentary: achievements and future directions for intervention research in communication and autism spectrum disorders. J. Autism Dev. Disord. 30:393-98
37. Lunceford JK, Davidian M, Tsiatis AA. 2002. Estimation of survival distributions of treatment policies in two-stage randomization designs in clinical trials. Biometrics 58:48-57
38. Marlowe DB, Festinger DS, Arabian PL, Dugosh KL, Benasutti KM, et al. 2008. Adaptive interventions in drug court: a pilot experiment. Crim. Justice Rev. 33:343-60
39. McKay JR. 2005. Is there a case for extended interventions for alcohol and drug use disorders? Addiction 100:1594-610
40. McKay JR. 2009. Continuing care research: what we have learned and where we are going. J. Subst. Abuse Treat. 36:131-45
41. McKay JR, Van Horn DHA, Oslin DW, Lynch KG, Ivey M, et al. 2010. A randomized trial of extended telephone-based continuing care for alcohol dependence: within-treatment substance use outcomes. J. Consult. Clin. Psychol. 78:912-23
42. McLellan AT. 2002. Have we evaluated addiction treatment correctly? Implications from a chronic care perspective. Addiction 97:249-52
43. McLellan AT, Lewis DC, O'Brien CP, Kleber HD. 2000. Drug dependence, a chronic medical illness- implications for treatment, insurance, and outcomes evaluation. J. Am. Med. Assoc. 284:1689-95
44. Miller WR, Moyers TB, Arciniega LT, DiClemente C, LoCastro J, et al. 2003. A combined behavioral intervention for treating alcohol dependence. Alcohol Clin. Exp. Res. 27(Suppl. 5):113A
45. Murphy, SA. 2005. An experimental design for the development of adaptive treatment strategies. Stat. Med. 24:1455-81
46. Murphy SA, Collins LM, Rush AJ. 2007a. Customizing treatment to the patient: adaptive treatment strategies. Drug Alcohol Depend. 88(Suppl. 2):1-3
47. Murphy SA, Lynch KG, Oslin D, McKay JR, TenHave T. 2007b. Developing adaptive treatment strategies in substance abuse. Drug Alcohol Depend. 88(Suppl. 2):24-30
48. Murphy SA, van der Laan MJ, Robins JM, CPPRG. 2001. Marginal mean models for dynamic regimes. J. Am. Stat. Assoc. 96:1410-23
49. Nahum-Shani I, Qian M, Almirall D, Pelham WE, Gnagy B, et al. 2011a. Experimental design and primary data analysis methods for comparing adaptive interventions. Manuscript submitted
50. Nahum-Shani I, Qian M, Almirall D, Pelham WE,Gnagy B, et al. 2011b. Q-Learning: a data analysismethod for constructing adaptive interventions. Manuscript submitted
51. Orellana L, Rotnitzky A, Robins JM. 2010. Dynamic regime marginal structural mean models for estimation of optimal dynamic treatment regimes, Part II: proofs of results. Int. J. Biostat. 6(2):article 9
52. Oslin D. 2005. Managing Alcoholism in People Who Do Not Respond to Naltrexone (EXTEND). Bethesda, MD: Natl. Inst. ealth. http://clinicaltrials.gov/ ct2/show/NCT00115037?term=oslin&rank=8
53. Pelham WE, Fabiano GA. 2008. Evidence-based psychosocial treatments for attention deficit/hyperactivity disorder. J. Clin. Child Adolesc. Psychol. 37:184-214
54. Pelham WE, Gnagy EM, Greenslade KE, Milich R. 1992. Teacher ratings of DSM-III-R symptoms for the disruptive behavior disorders. School Psychol. Rev. 21:285-99
55. Pettinati HM, Weiss RD, Dundon W, Miller WR, Donovan D, et al. 2005. A structured approach to medical management: a psychosocial intervention to support pharmacotherapy in the treatment of alcohol dependence. J. Stud. Alcohol Suppl. 15:170-78
56. Pettinati HM, Weiss RD, Miller WR, Donovan D, Ernst DB, Rounsaville BJ. 2004. A clinical research guide for medically trained clinicians providing pharmacotherapy as part of the treatment for alcohol dependence. In Medical Management Treatment Manual COMBINE Monograph Series, Volume 2, ed. ME Mattson. Bethesda, MD: Natl. Inst. Alcohol Abuse Alcohol.
57. Pocock SJ. 1977. Group sequential methods in the design and analysis of clinical trials. Biometrika 64:191-99
58. Robins JM. 1986. A new approach to causal inference in mortality studies with sustained exposure periods- application to control of the healthy worker survivor effect. Comput. Math. Appl. 14:1393-512
59. Robins JM, Hernán MA, Brumback B. 2000. Marginal structural models and causal inference in epidemiology. Epidemiology 11:550-60
60. Robins JM, Orellana L, Rotnitzky A. 2008. Estimation and extrapolation of optimal treatment and testing strategies. Stat. Med. 27(23):4678-721
61. Rubin DB. 1974. Estimating causal effects of treatments in randomized and nonrandomized studies. J. Educ. Psychol. 66:688-701
62. Rubin DB. 2007. The design versus the analysis of observational studies for causal effects: parallels with the design of randomized trials. Stat. Med. 26:20-36
63. Rush AJ, Fava M, Wisniewski SR, Lavori PW, Trivedi MH, et al. 2004. Sequenced treatment alternatives to relieve depression (STAR D): rationale and design. Control. Clin. Trials 25:119-42
64. Rush AJ, Trivedi MH, Wisniewski SR, Nierenberg AA, Stewart JW, et al. 2006. Acute and longer-term outcomes in depressed outpatients requiring one or several treatment steps: a STAR D report. Am. J. Psychiatry 163:1905-17
65. SAS Inst. 2008. SAS/STAT 9.2 User's Guide. Cary, NC: SAS Inst.
66. Schneider LS, Ismail MS,DagermanK,Davis S, Olin J, et al. 2003. Clinical antipsychotic trials of intervention effectiveness (CATIE): Alzheimer's disease trial. Schizophr. Bull. 29:57-72
67. Sobell MB, Sobell LC. 2000. Stepped care as a heuristic approach to the treatment of alcohol problems. J. Consult. Clin. Psychol. 68:573-79
68. Stone RM, Berg DT, George SL, Dodge RK, Paciucci PA, et al. 1995. Granulocyte-macrophage colonystimulating factor after initial chemotherapy for elderly patients with primary acute myelogenous leukemia. N. Engl. J. Med. 332:1671-77
69. Stroup TS,McEvoy JP, Swartz MS, ByerlyMJ, Glick ID, et al. 2003. TheNational Institute of Mental Health Clinical Antipsychotic Trials of Intervention Effectiveness (CATIE) project: schizophrenia trial design and protocol development. Schizophr. Bull. 29:15-31
70. StulzN,GallopR, LutzW,Wrenn GL,Crits-Christoph P. 2010. Examining differential effects of psychosocial treatments for cocaine dependence: an application of latent trajectory analyses. Drug Alcohol. Depend. 106:164-72
71. Tannir NM2010. Sequential Two-Agent Assessment in Renal Cell Carcinoma Therapy. Bethesda, MD: Natl. Inst. Health. http://clinicaltrials.gov/show/ NCT01217931
72. Thall PF, Logothetis C, Pagliaro LC, Wen SJ, Brown MA, et al. 2007a. Adaptive therapy for androgenindependent prostate cancer: a randomized selection trial of four regimens. J. Natl. Cancer Inst. 99:1613-22
73. Thall PF, SungHG, EsteyEH. 2002. Selecting therapeutic strategies based on efficacy and death in multicourse clinical trials. J. Am. Stat. Assoc. 97:29-39
74. Thall PF, Wathen JK. 2005. Covariate-adjusted adaptive randomization in a sarcoma trial with multi-stage treatments. Stat. Med. 24:1947-64
75. Thall PF, Wooten LH, Logothetis CJ, Millikan RE, Tannir NM. 2007b. Bayesian and frequentist two-stage treatment strategies based on sequential failure times subject to interval censoring. Stat.Med. 26:4687-702
76. Tummarello D, Mari D, Graziano F, Isidori P, Cetto G, et al. 1997. A randomized, controlled phase III study of cyclophosphamide, doxorubicin, and vincristine with etoposide (CAV-E) or teniposide (CAVT), followed by recombinant interferon-alpha maintenance therapy or observation, in small cell lung carcinoma patients with complete responses. Cancer 80:2222-29
77. Van Buuren S, Groothuis-Oudshoorn K. 2010. MICE: multivariate imputation by chained equations in R. J. Stat. Softw. In press
78. Wahed AS, Tsiatis AA. 2004. Optimal estimator for the survival distribution and related quantities for treatment policies in two-stage randomization designs in clinical trials. Biometrics 60:124-33
79. Wahed AS, Tsiatis AA. 2006. Semiparametric efficient estimation of survival distributions in two-stage randomisation designs in clinical trials with censored data. Biometrika 93:163-77
80. Weisz JR, Chu BC, Polo AJ. 2004. Treatment dissemination and evidence-based practice: strengthening intervention through clinician-researcher collaboration. Clin. Psychol. Sci. Pract. 11:300-7
81. Wigal SB, Wigal T, Schuck S, Brams M, Williamson D, et al. 2011. Academic, behavioral, and cognitive effects of OROS (R) methylphenidate on older children with attention deficit/hyperactivity disorder. J. Child Adolesc. Psychopharmacol. 21:121-31
82. Zhou X, Liu SY, Kim ES, Herbst RS, Lee JL. 2008. Bayesian adaptive design for targeted therapy development in lung cancer-a step toward personalized medicine. Clin. Trials 5:181-93