Combining single patient (N-of-1) trials to estimate population treatment effects and to evaluate individual patient responses to treatment
Title
Combining single patient (N-of-1) trials to estimate population treatment effects and to evaluate individual patient responses to treatment
Creator
Zucker DR; Schmid CH; McIntosh MW; D'Agostino RB; Selker HP; Lau J
Identifier
Publisher
Journal Of Clinical Epidemiology
Date
1997
Subject
Humans; Outcome Assessment (Health Care); Research Design; Antidepressive Agents; Cross-Over Studies; Models; Statistical; Chronic disease; Amitriptyline/therapeutic use; Bayes Theorem; Fibromyalgia/drug therapy; Randomized Controlled Trials as Topic/statistics & numerical data; Tricyclic
Description
When treating individual patients, physicians may face difficulties using the evidence from center-based randomized control trials (RCTs) due to limitations in these studies generalizability. Therefore, they often perform their own "informal" tests of treatment effectiveness. Single patient ("N-of-1") trials provide a structured design for more rigorous assessment of medical treatments of chronic diseases, but are applied only to the index patient. We present a hierarchical Bayesian random effects model to combine N-of-1 studies to obtain an estimate of treatment effectiveness for the population and to use this population information to aid in the evaluation of an individual patient's trial results. The model's treatment effect estimates are adjustments between the population estimate and the individual's observed results. This adjustment is based upon the within-patient and between-patient heterogeneity. We demonstrate this patient-focused method using published data from 23 N-of-1 trial results comparing amitriptyline and placebo for the treatment of fibromyalgia.
1997
Rights
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
Type
Journal Article
Citation List Month
Backlog
URL Address
Citation
Zucker DR; Schmid CH; McIntosh MW; D'Agostino RB; Selker HP; Lau J, “Combining single patient (N-of-1) trials to estimate population treatment effects and to evaluate individual patient responses to treatment,” Pediatric Palliative Care Library, accessed December 4, 2023, https://pedpalascnetlibrary.omeka.net/items/show/12193.