Literature DB >> 21695041

The n-of-1 clinical trial: the ultimate strategy for individualizing medicine?

Elizabeth O Lillie1, Bradley Patay, Joel Diamant, Brian Issell, Eric J Topol, Nicholas J Schork.   

Abstract

N-of-1 or single subject clinical trials consider an individual patient as the sole unit of observation in a study investigating the efficacy or side-effect profiles of different interventions. The ultimate goal of an n-of-1 trial is to determine the optimal or best intervention for an individual patient using objective data-driven criteria. Such trials can leverage study design and statistical techniques associated with standard population-based clinical trials, including randomization, washout and crossover periods, as well as placebo controls. Despite their obvious appeal and wide use in educational settings, n-of-1 trials have been used sparingly in medical and general clinical settings. We briefly review the history, motivation and design of n-of-1 trials and emphasize the great utility of modern wireless medical monitoring devices in their execution. We ultimately argue that n-of-1 trials demand serious attention among the health research and clinical care communities given the contemporary focus on individualized medicine.

Entities:  

Year:  2011        PMID: 21695041      PMCID: PMC3118090          DOI: 10.2217/pme.11.7

Source DB:  PubMed          Journal:  Per Med        ISSN: 1741-0541            Impact factor:   2.512


  76 in total

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2.  The path to personalized medicine.

Authors:  Margaret A Hamburg; Francis S Collins
Journal:  N Engl J Med       Date:  2010-06-15       Impact factor: 91.245

3.  The n-of-1 randomized controlled trial: clinical usefulness. Our three-year experience.

Authors:  G H Guyatt; J L Keller; R Jaeschke; D Rosenbloom; J D Adachi; M T Newhouse
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4.  Research agenda. Opportunities for research and NIH.

Authors:  Francis S Collins
Journal:  Science       Date:  2010-01-01       Impact factor: 47.728

5.  Using N-of-1 trials to improve patient management and save costs.

Authors:  Paul A Scuffham; Jane Nikles; Geoffrey K Mitchell; Michael J Yelland; Norma Vine; Christopher J Poulos; Peter I Pillans; Guy Bashford; Chris del Mar; Philip J Schluter; Paul Glasziou
Journal:  J Gen Intern Med       Date:  2010-04-13       Impact factor: 5.128

6.  A pilot study of ondansetron plus metopimazine vs. ondansetron monotherapy in children receiving highly emetogenic chemotherapy: a Bayesian randomized serial N-of-1 trials design.

Authors:  P C Nathan; G Tomlinson; L L Dupuis; M L Greenberg; S Ota; U Bartels; B M Feldman
Journal:  Support Care Cancer       Date:  2005-07-29       Impact factor: 3.603

7.  Prioritising drugs for single patient (n-of-1) trials in palliative care.

Authors:  J Nikles; G Mitchell; J Walters; J Hardy; P Good; D Rowett; T Shelby-James; D Currow
Journal:  Palliat Med       Date:  2009-07-15       Impact factor: 4.762

Review 8.  Genomic and personalized medicine: foundations and applications.

Authors:  Geoffrey S Ginsburg; Huntington F Willard
Journal:  Transl Res       Date:  2009-10-01       Impact factor: 7.012

Review 9.  Clinically available pharmacogenomics tests.

Authors:  D A Flockhart; T Skaar; D S Berlin; T E Klein; A T Nguyen
Journal:  Clin Pharmacol Ther       Date:  2009-04-15       Impact factor: 6.875

Review 10.  Circulating endothelial cells and endothelial progenitors as surrogate biomarkers in vascular dysfunction.

Authors:  Huakang Wu; Hongli Chen; Peter C Hu
Journal:  Clin Lab       Date:  2007       Impact factor: 1.138

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  184 in total

Review 1.  Non-celiac gluten sensitivity: Time for sifting the grain.

Authors:  Luca Elli; Leda Roncoroni; Maria Teresa Bardella
Journal:  World J Gastroenterol       Date:  2015-07-21       Impact factor: 5.742

Review 2.  A systems approach to clinical oncology uses deep phenotyping to deliver personalized care.

Authors:  James T Yurkovich; Qiang Tian; Nathan D Price; Leroy Hood
Journal:  Nat Rev Clin Oncol       Date:  2019-10-16       Impact factor: 66.675

3.  Patient preferences for personalized (N-of-1) trials: a conjoint analysis.

Authors:  Nathalie Moise; Dallas Wood; Ying Kuen K Cheung; Naihua Duan; Tara St Onge; Joan Duer-Hefele; Tiffany Pu; Karina W Davidson; Ian M Kronish
Journal:  J Clin Epidemiol       Date:  2018-05-30       Impact factor: 6.437

Review 4.  Taking Systems Medicine to Heart.

Authors:  Kalliopi Trachana; Rhishikesh Bargaje; Gustavo Glusman; Nathan D Price; Sui Huang; Leroy E Hood
Journal:  Circ Res       Date:  2018-04-27       Impact factor: 17.367

5.  Nocturnal continuous glucose and sleep stage data in adults with type 1 diabetes in real-world conditions.

Authors:  Stephanie Feudjio Feupe; Patrick F Frias; Sara C Mednick; Elizabeth A McDevitt; Nathaniel D Heintzman
Journal:  J Diabetes Sci Technol       Date:  2013-09-01

6.  N-of-1 Randomized Trials of Ultra-Micronized Palmitoylethanolamide in Older Patients with Chronic Pain.

Authors:  Federico Germini; Anna Coerezza; Luca Andreinetti; Alessandro Nobili; Paolo Dionigi Rossi; Daniela Mari; Gordon Guyatt; Maura Marcucci
Journal:  Drugs Aging       Date:  2017-12       Impact factor: 3.923

7.  TummyTrials: A Feasibility Study of Using Self-Experimentation to Detect Individualized Food Triggers.

Authors:  Ravi Karkar; Jessica Schroeder; Daniel A Epstein; Laura R Pina; Jeffrey Scofield; James Fogarty; Julie A Kientz; Sean A Munson; Roger Vilardaga; Jasmine Zia
Journal:  Proc SIGCHI Conf Hum Factor Comput Syst       Date:  2017-05-02

8.  Tumour heterogeneity in the clinic.

Authors:  Philippe L Bedard; Aaron R Hansen; Mark J Ratain; Lillian L Siu
Journal:  Nature       Date:  2013-09-19       Impact factor: 49.962

9.  Therapeutic horseback riding outcomes of parent-identified goals for children with autism spectrum disorder: an ABA' multiple case design examining dosing and generalization to the home and community.

Authors:  Margo B Holm; Joanne M Baird; Young Joo Kim; Kuwar B Rajora; Delma D'Silva; Lin Podolinsky; Carla Mazefsky; Nancy Minshew
Journal:  J Autism Dev Disord       Date:  2014-04

Review 10.  Building a personalized medicine infrastructure at a major cancer center.

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Journal:  J Clin Oncol       Date:  2013-04-15       Impact factor: 44.544

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