Statistics in Medicine

Estimating heterogeneous treatment effects for latent subgroups in observational studies

Journal Article

Individuals may vary in their responses to treatment, and identification of subgroups differentially affected by a treatment is an important issue in medical research. The risk of misleading subgroup analyses has become well known, and some exploratory analyses can be helpful in clarifying how covariates potentially interact with the treatment. Motivated by a real data study of pediatric kidney transplant, we consider a semiparametric Bayesian latent model and examine its utility for an exploratory subgroup effect analysis using secondary data. The proposed method is concerned with a clinical setting where the number of subgroups is much smaller than that of potential predictors and subgroups are only latently associated with observed covariates. The semiparametric model is flexible in capturing the latent structure driven by data rather than dictated by parametric modeling assumptions. Since it is difficult to correctly specify the conditional relationship between the response and a large number of confounders in modeling, we use propensity score matching to improve the model robustness by balancing the covariates distribution. Simulation studies show that the proposed analysis can find the latent subgrouping structure and, with propensity score matching adjustment, yield robust estimates even when the outcome model is misspecified. In the real data analysis, the proposed analysis reports significant subgroup effects on steroid avoidance in kidney transplant patients, whereas standard proportional hazards regression analysis does not.

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Published features on are checked for statistical accuracy by a panel from the European Network for Business and Industrial Statistics (ENBIS)   to whom Wiley and express their gratitude. This panel are: Ron Kenett, David Steinberg, Shirley Coleman, Irena Ograjenšek, Fabrizio Ruggeri, Rainer Göb, Philippe Castagliola, Xavier Tort-Martorell, Bart De Ketelaere, Antonio Pievatolo, Martina Vandebroek, Lance Mitchell, Gilbert Saporta, Helmut Waldl and Stelios Psarakis.