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

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

Pages

401-410

Issue

4

Volume

50

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 September 21, 2021, https://pedpalascnetlibrary.omeka.net/items/show/12193.

Social Bookmarking