{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2023,12,11]],"date-time":"2023-12-11T14:43:27Z","timestamp":1702305807467},"reference-count":15,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2016,9,19]],"date-time":"2016-09-19T00:00:00Z","timestamp":1474243200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2016,9,19]],"date-time":"2016-09-19T00:00:00Z","timestamp":1474243200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Sci Rep"],"abstract":"<jats:title>Abstract<\/jats:title><jats:p>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 \u201ccensoring by death\u201d 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.<\/jats:p>","DOI":"10.1038\/srep33431","type":"journal-article","created":{"date-parts":[[2016,9,19]],"date-time":"2016-09-19T10:06:41Z","timestamp":1474279601000},"update-policy":"http:\/\/dx.doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Estimating the palliative effect of percutaneous endoscopic gastrostomy in an observational registry using principal stratification and generalized propensity scores"],"prefix":"10.1038","volume":"6","author":[{"given":"Pallavi S.","family":"Mishra-Kalyani","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Brent A.","family":"Johnson","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jonathan D.","family":"Glass","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Qi","family":"Long","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2016,9,19]]},"reference":[{"key":"BFsrep33431_CR1","unstructured":"Gelinas, D. F. & Miller, R. G. A treatable disease: a guide to the management of amyotrophic lateral sclerosis. Amyotrophic lateral sclerosis 405\u2013421 (2000)."},{"key":"BFsrep33431_CR2","unstructured":"Procaccini, N. J. & Nemergut, E. C. Percutaneous endoscopic gastrostomy in the patient with amyotrophic lateral sclerosis: Risk vs benefit? Pract. Gastroenterology 24 (2008)."},{"key":"BFsrep33431_CR3","doi-asserted-by":"publisher","first-page":"21","DOI":"10.1111\/j.0006-341X.2002.00021.x","volume":"58","author":"CE Frangakis","year":"2002","unstructured":"Frangakis, C. E. & Rubin, D. B. Principal stratification in causal inference. Biometrics 58(1), 21\u201329 (2002).","journal-title":"Biometrics"},{"key":"BFsrep33431_CR4","doi-asserted-by":"publisher","first-page":"353","DOI":"10.3102\/10769986028004353","volume":"28","author":"JL Zhang","year":"2003","unstructured":"Zhang, J. L. & Rubin, D. B. Estimation of causal effects via principal stratification when some outcomes are truncated by \u201cdeath\u201d. J. of Educ. and Behav. Stat. 28(4), 353\u2013368 (2003).","journal-title":"J. of Educ. and Behav. Stat."},{"key":"BFsrep33431_CR5","doi-asserted-by":"crossref","unstructured":"Zhang, J. L., Rubin, D. B. & Mealli, F. Likelihood-based analysis of causal effects of job-training programs using principal stratification. J. Amer. Statist. Assoc. 104(485) (2009).","DOI":"10.1198\/jasa.2009.0012"},{"key":"BFsrep33431_CR6","doi-asserted-by":"publisher","first-page":"949","DOI":"10.1002\/sim.4472","volume":"31","author":"S Schwartz","year":"2012","unstructured":"Schwartz, S., Li, F. & Reiter, J. P. Sensitivity analysis for unmeasured confounding in principal stratification settings with binary variables. Stat. in Medicine 31(10), 949\u2013962 (2012).","journal-title":"Stat. in Medicine"},{"key":"BFsrep33431_CR7","doi-asserted-by":"publisher","first-page":"41","DOI":"10.1093\/biomet\/70.1.41","volume":"70","author":"PR Rosenbaum","year":"1983","unstructured":"Rosenbaum, P. R. & Rubin, D. B. The central role of the propensity score in observational studies for causal effects. Biometrika 70(1), 41\u201355 (1983).","journal-title":"Biometrika"},{"key":"BFsrep33431_CR8","doi-asserted-by":"publisher","first-page":"854","DOI":"10.1198\/016214504000001187","volume":"99","author":"K Imai","year":"2004","unstructured":"Imai, K. & Van Dyk, D. A. Causal inference with general treatment regimes: Generalizing the propensity score. J. Amer. Statist. Assoc. 99(467), 854\u2013866 (2004).","journal-title":"J. Amer. Statist. Assoc."},{"key":"BFsrep33431_CR9","doi-asserted-by":"crossref","unstructured":"Hirano, K. & Imbens, G. W. The propensity score with continuous treatments. Missing Data and Bayesian Methods in Practice: Contribution by Donald Rubin\u2019s Statistical Family 73\u201384 (2004).","DOI":"10.1002\/0470090456.ch7"},{"key":"BFsrep33431_CR10","doi-asserted-by":"publisher","first-page":"2857","DOI":"10.1002\/sim.3669","volume":"28","author":"B Jo","year":"2009","unstructured":"Jo, B. & Stuart, E. A. On the use of propensity scores in principal causal effect estimation. Stat. in Medicine 28(23), 2857\u20132875 (2009).","journal-title":"Stat. in Medicine"},{"key":"BFsrep33431_CR11","first-page":"591","volume":"75","author":"DB Rubin","year":"1980","unstructured":"Rubin, D. B. Randomization analysis of experimental data: The Fisher randomization test (comment). J. Amer. Statist. Assoc. 75(371), 591\u2013593 (1980).","journal-title":"J. Amer. Statist. Assoc."},{"key":"BFsrep33431_CR12","doi-asserted-by":"publisher","first-page":"945","DOI":"10.1080\/01621459.1986.10478354","volume":"81","author":"PW Holland","year":"1986","unstructured":"Holland, P. W. Statistics and causal inference\u2019. J. Amer. Statist. Assoc. 81(396), 945\u2013960 (1986).","journal-title":"J. Amer. Statist. Assoc."},{"key":"BFsrep33431_CR13","unstructured":"Zhao, S., van Dyk, D. A. & Imai, K. Causal inference in observational studies with non-binary treatments."},{"key":"BFsrep33431_CR14","doi-asserted-by":"publisher","first-page":"528","DOI":"10.1080\/01621459.1987.10478458","volume":"82","author":"MA Tanner","year":"1987","unstructured":"Tanner, M. A. & Wong, W. H. The calculation of posterior distributions by data augmentation. J. Amer. Statist. Assoc. 82(398), 528\u2013540 (1987).","journal-title":"J. Amer. Statist. Assoc."},{"key":"BFsrep33431_CR15","doi-asserted-by":"publisher","first-page":"263","DOI":"10.1111\/j.1541-0420.2012.01830.x","volume":"69","author":"CM Zigler","year":"2013","unstructured":"Zigler, C. M. et al. Model feedback in Bayesian propensity score estimation\u2019. Biometrics 69(1), 263\u2013273 (2013).","journal-title":"Biometrics"}],"container-title":["Scientific Reports"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.nature.com\/articles\/srep33431.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.nature.com\/articles\/srep33431","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.nature.com\/articles\/srep33431.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,1,5]],"date-time":"2023-01-05T06:55:01Z","timestamp":1672901701000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.nature.com\/articles\/srep33431"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2016,9,19]]},"references-count":15,"journal-issue":{"issue":"1","published-online":{"date-parts":[[2016,12,16]]}},"alternative-id":["BFsrep33431"],"URL":"https:\/\/doi.org\/10.1038\/srep33431","relation":{},"ISSN":["2045-2322"],"issn-type":[{"value":"2045-2322","type":"electronic"}],"subject":[],"published":{"date-parts":[[2016,9,19]]},"assertion":[{"value":"29 February 2016","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"26 August 2016","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"19 September 2016","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"The authors declare no competing financial interests.","order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"33431"}}