Up-front versus sequential randomizations for inference on adaptive treatment strategies

Statistics in Medicine

Volumn 31, Issue 9, 2012, Pages 812-830

  Ko, Jin H ^a   Wahed, Abdus S ^a  

^a UNIVERSITY OF PITTSBURGH GRADUATE SCHOOL OF PUBLIC HEALTH   (United States)

Author keywords

Adaptive treatment strategies; Inverse probability weighting; Sequential randomization; Two stage randomization design

Indexed keywords

ACCURACY; ARTICLE; COMPARATIVE EFFECTIVENESS; HEALTH CARE PLANNING; HUMAN; INTERMETHOD COMPARISON; MATHEMATICAL COMPUTING; MATHEMATICAL MODEL; MEDICAL DECISION MAKING; MOTIVATION; OUTCOME ASSESSMENT; PROBABILITY; PROCESS DEVELOPMENT; PROCESS MODEL; PROCESS OPTIMIZATION; RANDOMIZATION; RELIABILITY; SAMPLE SIZE; STRATEGIC PLANNING; SYSTEMATIC ERROR; TREATMENT PLANNING; TREND STUDY; VALIDATION PROCESS;

ANTINEOPLASTIC AGENTS; COMPUTER SIMULATION; HUMANS; LEUKEMIA; MONTE CARLO METHOD; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RESEARCH DESIGN; TREATMENT OUTCOME;

EID: 84859565126     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.4473     Document Type: Article

References (13)

1. Stone RM, Berg DT, George SL, Dodge RK, Paciucci PA, Schulman P, Lee EJ, Moore JO, Powell BL, Schiffer CA. Granulocytemacrophage colony-stimulating factor after initial chemotherapy for elderly patients with primary acute myelogenous leukemia. The New England Journal of Medicine 1994; 332:1671-1677. DOI: 10.1056/NEJM199506223322503.
2. Thall PF, Wooten LH, Logothetis CJ, Millikan RE, Tannir NM. Bayesian and frequentist two-stage treatment strategies based on sequential failure times subject to interval censoring. Statistics in Medicine 2007; 26:4687-4702. DOI: 10.1002/sim.2894.
3. Murphy SA. An experimental design for the development of adaptive treatment strategies. Statistics in Medicine 2005; 24:1455-1481. DOI: 10.1002/sim.2022.
4. Robins JM, Rotnitzky A, Zhao LP. Estimation of regression coefficients when some regressors are not always observed. Journal of the American Statistical Association 1994; 89:846-866. DOI: 10.2307/2290910.
5. Lunceford JK, Davidian M, Tsiatis AA. Estimation of survival distributions of treatment policies in two-stage randomization designs in clinical trials. Biometrics 2002; 58:48-57. DOI: 10.1111/j.0006-341X.2002.00048.x.
6. Wahed AS, Tsiatis AA. Optimal estimator for the survival distribution and related quantities for treatment policies in two-stage randomization designs in clinical trials. Biometrics 2004; 60:124-133. DOI: 10.1111/j.0006-341X.2004.00160.x.
7. Wolbers M, Helterbrand JD. Two-stage randomization designs in drug development. Statistics in Medicine 2008; 27:4161-4174. DOI: 10.1002/sim.3309.
8. Holland PW. Statistics and causal inference. Journal of the American Statistical Association 1986; 81:945-970. DOI: 10.2307/2289064.
9. Robins JM. A new approach to causal inference in mortality studies with sustained exposure periods-application to control of the healthy worker survivor effect. Mathematical Modeling 1986; 7:1393-1512. DOI: 10.1016/0270-0255(86)90088-6.
10. Cole SR, Frangakisb CE. The consistency statement in causal inference: a definition or an assumption? Epidemiology 2009; 20(1):3-5. DOI: 10.1097/EDE.0b013e31818ef366.
11. Robins JM. Association, causation, and marginal structural models. Synthese 1999; 121:151-179. DOI: 10.1023/A:1005285815569.
12. Stefanski LA, Boos DD. The calculus of M-estimation. The American Statisticion 2002; 56:29-38. DOI: 10.1198/000313002753631330.
13. Buyze J, Van Rompaye B, Goetghebur E. Designing a sequentially randomized study with adherence enhancing interventions for diabetes patients. Statistics in Medicine 2010; 29:1114-1126. DOI: 10.1002/sim.3856.