Evaluation of person-level heterogeneity of treatment effects in published multiperson N-of-1 studies: systematic review and reanalysis. Issue 5 (26th May 2018)

• Record Type: Journal Article
• Authors: Raman, Gowri; Balk, Ethan M; Lai, Lana; Shi, Jennifer; Chan, Jeffrey; Lutz, Jennifer S; Dubois, Robert W; Kravitz, Richard L; Kent, David M
• Is Part Of: BMJ open. Volume 8:Issue 5(2018)
• Publication Date: 2018-05-26
• Subjects: perseonalized medicine -- N-of-1 studies -- systematic review -- heterogeneity of treatment effect; Medicine -- Research -- Periodicals; 610.72
• DOI: 10.1136/bmjopen-2017-017641
• Languages: English
• ISSNs: 2044-6055

Abstract

Abstract : Objective: Individual patients with the same condition may respond differently to similar treatments. Our aim is to summarise the reporting of person-level heterogeneity of treatment effects (HTE) in multiperson N-of-1 studies and to examine the evidence for person-level HTE through reanalysis. Study design: Systematic review and reanalysis of multiperson N-of-1 studies. Data sources: Medline, Cochrane Controlled Trials, EMBASE, Web of Science and review of references through August 2017 for N-of-1 studies published in English. Study selection: N-of-1 studies of pharmacological interventions with at least two subjects. Data synthesis: Citation screening and data extractions were performed in duplicate. We performed statistical reanalysis testing for person-level HTE on all studies presenting person-level data. Results: We identified 62 multiperson N-of-1 studies with at least two subjects. Statistical tests examining HTE were described in only 13 (21%), of which only two (3%) tested person-level HTE. Only 25 studies (40%) provided person-level data sufficient to reanalyse person-level HTE. Reanalysis using a fixed effect linear model identified statistically significant person-level HTE in 8 of the 13 studies (62%) reporting person-level treatment effects and in 8 of the 14 studies (57%) reporting person-level outcomes. Conclusions: Our analysis suggests that person-level HTE is common and often substantial. Reviewed studies had incomplete information on person-level treatment effects and their variation. Improved assessment and reporting of person-level treatment effects in multiperson N-of-1 studies are needed.