title: Estimating the palliative effect of percutaneous endoscopic gastrostomy in an observational registry using principal stratification and generalized propensity scores.
abstract: Clinical disease registries offer a rich collection of valuable patient information but also pose challenges that require special care and attention in statistical analyses. The goal of this paper is to propose a statistical framework that allows for estimating the effect of surgical insertion of a percutaneous endogastrostomy (PEG) tube for patients living with amyotrophic lateral sclerosis (ALS) using data from a clinical registry. Although all ALS patients are informed about PEG, only some patients agree to the procedure which, leads to the potential for selection bias. Assessing the effect of PEG is further complicated by the aggressively fatal disease, such that time to death competes directly with both the opportunity to receive PEG and clinical outcome measurements. Our proposed methodology handles the "censoring by death" phenomenon through principal stratification and selection bias for PEG treatment through generalized propensity scores. We develop a fully Bayesian modeling approach to estimate the survivor average causal effect (SACE) of PEG on BMI, a surrogate outcome measure of nutrition and quality of life. The use of propensity score methods within the principal stratification framework demonstrates a significant and positive effect of PEG treatment, particularly when time of treatment is included in the treatment definition.
authors.affiliation: Food and Drug Administration, Office of Biostatistics, Silver Spring, 20903, USA.
authors.forename: Pallavi S
authors.initials: PS
authors.lastname: Mishra-Kalyani
authors.affiliation: University of Rochester, Department of Biostatistics and Computational Biology, Rochester, 14642, USA.
authors.forename: Brent A
authors.initials: BA
authors.lastname: Johnson
authors.affiliation: Emory University, Department of Neurology, Atlanta, 30322, USA.
authors.forename: Jonathan D
authors.initials: JD
authors.lastname: Glass
authors.affiliation: Emory University, Department of Biostatistics and Bioinformatics, Atlanta, 30322, USA.
authors.forename: Qi
authors.initials: Q
authors.lastname: Long
mesh_terms: D000690:Amyotrophic Lateral Sclerosis / Q000601:surgery; D015992:Body Mass Index; D004724:Endoscopy / Q000009:adverse effects; D005774:Gastrostomy / Q000009:adverse effects; D006801:Humans; D016013:Likelihood Functions; D010166:Palliative Care; D057216:Propensity Score; D012042:Registries