Estimating the palliative effect of percutaneous endoscopic gastrostomy in an observational registry using principal stratification and generalized propensity scores.

Publisher: Nature Publishing Group Country of Publication: England NLM ID: 101563288 Publication Model: Electronic Cited Medium: Internet ISSN: 2045-2322 (Electronic) Linking ISSN: 20452322 NLM ISO Abbreviation: Sci Rep Subsets: MEDLINE

Original Publication: London : Nature Publishing Group, copyright 2011-

Endoscopy*/adverse effects ; Gastrostomy*/adverse effects ; Palliative Care* ; Propensity Score* ; Registries*; Amyotrophic Lateral Sclerosis/surgery ; Body Mass Index ; Humans ; Likelihood Functions

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.

Biometrics. 2013 Mar;69(1):263-73. (PMID: 23379793)
Stat Methods Med Res. 2020 Mar;29(3):709-727. (PMID: 32186266)
Stat Med. 2009 Oct 15;28(23):2857-75. (PMID: 19610131)
Stat Med. 2012 May 10;31(10):949-62. (PMID: 22362635)
Biometrics. 2002 Mar;58(1):21-9. (PMID: 11890317)

R21 NS091630 United States NS NINDS NIH HHS

Date Created: 20160920 Date Completed: 20180604 Latest Revision: 20240325

20250114

PMC5027570

10.1038/srep33431

27640365