Salima Punja1, Cecilia Bukutu2, Larissa Shamseer3, Margaret Sampson4, Lisa Hartling5, Liana Urichuk6, Sunita Vohra7. 1. Department of Pediatrics, University of Alberta, 1702 College Plaza, 8215-112 Street NW, Edmonton, Alberta T6G 2C8, Canada. 2. Department of Pediatrics, University of Alberta, 1702 College Plaza, 8215-112 Street NW, Edmonton, Alberta T6G 2C8, Canada; Knowledge Utilization Studies Program, Faculty of Nursing, University of Alberta, 3rd Floor Edmonton Clinic Health Academy, Edmonton, Alberta T6G 1C9, Canada. 3. Clinical Epidemiology Program, Ottawa Hospital Research Institute, Center for Practice Changing Research Building, 501 Smyth Road, PO Box 201B, Ottawa, Ontario K1H 8L6, Canada. 4. Department of Library Services, Children's Hospital of Eastern Ontario, 401 Smyth Road, Ottawa, Ontario K1H 8L1, Canada. 5. Alberta Research Centre for Health Evidence, Department of Pediatrics, University of Alberta, 4-472 Edmonton Clinic Health Academy, 11405-87 Avenue, Edmonton, Alberta T6G 1C9, Canada. 6. Information & Evaluation Services for Addiction & Mental Health, Addiction and Mental Health, Alberta Health Services-Edmonton Zone, 9942 108 Street, Edmonton, Alberta T5K 2J5, Canada. 7. Complementary and Alternative Research and Education Program (CARE), Department of Pediatrics, University of Alberta. Electronic address: svohra@ualberta.ca.
Abstract
OBJECTIVES: To summarize the methods of design, analysis, and meta-analysis used in N-of-1 trials. STUDY DESIGN AND SETTING: Electronic search for English language articles published from 1950 to 2013. N-of-1 trials were selected if they followed an ABAB design and if they assessed a health intervention for a medical condition. Elements of design, analysis, and meta-analysis were extracted. RESULTS: We included 100 reports representing 1,995 participants. N-of-1 trials have been conducted in over 50 health conditions. Most reports incorporated the use of elements that maintain methodological rigor, including randomization, blinding, and formal outcome assessment; however, many failed to address trial registration, funding source, and adverse events. Most reports statistically analyzed individual N-of-1 trials; however, only a small proportion of included series meta-analyzed their results. CONCLUSIONS: N-of-1 trials have the ability to assess treatment response in individual participants and can be used for a variety of health interventions for a wide range of medical conditions in both clinical and research settings. Considerable heterogeneity exists in the methods used in N-of-1 trials.
OBJECTIVES: To summarize the methods of design, analysis, and meta-analysis used in N-of-1 trials. STUDY DESIGN AND SETTING: Electronic search for English language articles published from 1950 to 2013. N-of-1 trials were selected if they followed an ABAB design and if they assessed a health intervention for a medical condition. Elements of design, analysis, and meta-analysis were extracted. RESULTS: We included 100 reports representing 1,995 participants. N-of-1 trials have been conducted in over 50 health conditions. Most reports incorporated the use of elements that maintain methodological rigor, including randomization, blinding, and formal outcome assessment; however, many failed to address trial registration, funding source, and adverse events. Most reports statistically analyzed individual N-of-1 trials; however, only a small proportion of included series meta-analyzed their results. CONCLUSIONS: N-of-1 trials have the ability to assess treatment response in individual participants and can be used for a variety of health interventions for a wide range of medical conditions in both clinical and research settings. Considerable heterogeneity exists in the methods used in N-of-1 trials.
Authors: Elizabeth M Berry-Kravis; Lothar Lindemann; Aia E Jønch; George Apostol; Mark F Bear; Randall L Carpenter; Jacqueline N Crawley; Aurore Curie; Vincent Des Portes; Farah Hossain; Fabrizio Gasparini; Baltazar Gomez-Mancilla; David Hessl; Eva Loth; Sebastian H Scharf; Paul P Wang; Florian Von Raison; Randi Hagerman; Will Spooren; Sébastien Jacquemont Journal: Nat Rev Drug Discov Date: 2017-12-08 Impact factor: 84.694