Evidence map›Paper›PMID 21695041›Full record

ArticlePersonalized medicine2011

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

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

3 registry-linked trialsAbstract read
In one paragraph

Article in Personalized medicine, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 3 registered trials, which are not on this map. Cited by 305 papers, 4 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
305citing papers in PubMed, 4 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT03351569 phase3unknown statusnot on this mapstarted 2015, after this paper: background citation

Intravenous Immunoglobulin for Unverricht-Lundborg Disease: Single-patient Trial.

TypeinterventionalSponsorAzienda Socio Sanitaria Territoriale di MantovaRan2015 to 2017Enrolled1ConditionsUnverricht-Lundborg DiseaseArmsIntravenous immunoglobulin
NCT03949959 nacompletednot on this mapstarted 2019, after this paper: background citation

Effectiveness of Conservative Multimodal Physiotherapy in Chronic Whiplash-associated Disorders With Facet-mediated Pain Undergoing Platelet Rich Plasma (PRP) Treatment: a Series of Single Case Experimental Designs (SCEDs).

TypeinterventionalSponsorUniversity of CalgaryRan2019 to 2021Enrolled6ConditionsWhiplash Injuries, Neck Injuries, Platelet Rich PlasmaArmsMultimodal physiotherapy
NCT06821776 narecruitingnot on this mapstarted 2025, after this paper: background citation

N-of-1 Trial Comparing Prolonged Gastric Feeding to Transpyloric Feeding in Infants with Severe Bronchopulmonary Dysplasia

TypeinterventionalSponsorLe Bonheur Children's HospitalRan2025 to 2030Enrolled25ConditionsBronchopulmonary Dysplasia, Feeding IntoleranceArmsGastric feeding, Transpyloric feeding
3 · Its place in the literature

Who cites it

305 citing papers in PubMed, 4 syntheses or guidelines pooled it.

  1. Pooled it
  2. Pooled it
  3. Pooled it
  4. Pooled it
  5. Trial
  6. Trial
  7. Trial
  8. Trial
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  10. Trial
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  12. A Randomized Placebo-ControlledCanadian journal of gastroenterology & hepatology · 2019
    Trial
  13. Article
  14. Review
  15. Article
  16. Review
  17. Randomised controlled trials and iNPH.Fluids and barriers of the CNS · 2026
    Article
  18. Article
  19. Review
  20. Article

245 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors.

Elizabeth O LillieScripps Health, La Jolla, CA 92037, USA.
Bradley Patay
Joel Diamant
Brian Issell
Eric J Topol
Nicholas J Schork

Funding

Systems BiologyU19AG023122 · NIA · TRANSLATIONAL GENOMICS RESEARCH INST · PI NICHOLAS Joseph SCHORK · 2004 to 2026
$102.6M
SCRIPPS TRANSLATIONAL SCIENCE INSTITUTE (UL1)UL1RR025774 · NCRR · SCRIPPS RESEARCH INSTITUTE, THE · PI TOPOL, ERIC JEFFREY · 2008 to 2011
$16.3M
Substance Abuse & Behavioral Disinhibition: Integrating Genes & EnvironmentU01DA024417 · NIDA · UNIVERSITY OF MINNESOTA · PI IACONO, WILLIAM G. · 2007 to 2010
$10.8M
TARGETING GENETIC PATHWAYS FOR BRAIN OVERGROWTH IN AUTISM SPECTRUM DISORDERSP50MH081755 · NIMH · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI COURCHESNE, ERIC · 2007 to 2013
$10.3M
TopologyU54CA143906 · NCI · UNIVERSITY OF SOUTHERN CALIFORNIA · PI KUHN, PETER · 2009 to 2014
$10.2M
Genomic Association Study of Bipolar DisorderR01MH078151 · NIMH · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI KELSOE, JOHN R. · 2007 to 2009
$5.5M
Genome Wide Association of Coronary Artery Disease and Related Risk FactorsR01HL089655 · NHLBI · MEDICAL COLLEGE OF WISCONSIN · PI BROECKEL, ULRICH · 2008 to 2012
$3.8M
Studying the Biology and Behavior of Autism at 1-Year: The Well-Baby Check-Up AppR01MH080134 · NIMH · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI PIERCE, KAREN L · 2008 to 2017
$3.3M
The Bipolar Genome StudyR01MH094483 · NIMH · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI CRAIG, DAVID W, EDENBERG, HOWARD J · 2012 to 2014
$2.2M
Circadian rhythm genes and sleep disturbances in the elderlyR01AG030474 · NIA · CALIFORNIA PACIFIC MED CTR RES INSTITUTE · PI TRANAH, GREGORY J. · 2008 to 2010
$1.4M
CNS HIV ANTI-RETROVIRAL THERAPY EFFECTS RESEARCH /CHARTER/ -278022005-278022005N01MH022005 · NIMH · UNIVERSITY OF CA -SAN DIEGO · PI GRANT, IGOR · 2003 to 2006
–
NCI NIH HHS U54 CA143906NCRR NIH HHS UL1 RR025774NHLBI NIH HHS R01 HL089655NIA NIH HHS R01 AG030474NIA NIH HHS U19 AG023122NIDA NIH HHS U01 DA024417NIMH NIH HHS N01 MH022005NIMH NIH HHS P50 MH081755NIMH NIH HHS R01 MH078151NIMH NIH HHS R01 MH080134NIMH NIH HHS R01 MH094483
6 · The paper itself

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.

Identifiers

PMID21695041
PMCPMC3118090

What OpenQuestion holds

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Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.