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A Case for n-of-1 Trials—Reply

A Case for n-of-1 Trials—Reply Letters tients had hypertension, which was similar to users of dipep- in treatment effect; three-quarters of participants had no differ- tidyl peptidase 4 inhibitors and glucagon-like peptide 1 recep- ence. Should this be interpreted as a failure of n-of-1 trials? No, tor agonists, but higher than users of other oral medications. just as a negative randomized clinical trial is not a “failure” of ran- Second, approximately 12% and 17% to 18% of patients were domized clinical trials. If 2 interventions offer no difference in classified as current smokers in the LEADER and CANVAS trials, outcome to patients, this is worth knowing. 3,4 respectively. The smoking status of patients was unknown Precision medicine is important because of heteroge- in the cohort trial. The prevalence of peripheral artery dis- neity of treatment effect. As such, n-of-1 trials determine which 3 4 ease was similar between the LEADER (17.6%) and CANVAS therapies are effective in specific individuals, assess whether (21%) trials, but this condition was not reported in baseline previously effective treatments are still effective (which is im- characteristics for the cohort trial. Finally, the history of pre- portant in chronic disease management), and offer a cost- vious amputations could have been http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png JAMA Internal Medicine American Medical Association

A Case for n-of-1 Trials—Reply

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Publisher
American Medical Association
Copyright
Copyright 2019 American Medical Association. All Rights Reserved.
ISSN
2168-6106
eISSN
2168-6114
DOI
10.1001/jamainternmed.2018.7180
Publisher site
See Article on Publisher Site

Abstract

Letters tients had hypertension, which was similar to users of dipep- in treatment effect; three-quarters of participants had no differ- tidyl peptidase 4 inhibitors and glucagon-like peptide 1 recep- ence. Should this be interpreted as a failure of n-of-1 trials? No, tor agonists, but higher than users of other oral medications. just as a negative randomized clinical trial is not a “failure” of ran- Second, approximately 12% and 17% to 18% of patients were domized clinical trials. If 2 interventions offer no difference in classified as current smokers in the LEADER and CANVAS trials, outcome to patients, this is worth knowing. 3,4 respectively. The smoking status of patients was unknown Precision medicine is important because of heteroge- in the cohort trial. The prevalence of peripheral artery dis- neity of treatment effect. As such, n-of-1 trials determine which 3 4 ease was similar between the LEADER (17.6%) and CANVAS therapies are effective in specific individuals, assess whether (21%) trials, but this condition was not reported in baseline previously effective treatments are still effective (which is im- characteristics for the cohort trial. Finally, the history of pre- portant in chronic disease management), and offer a cost- vious amputations could have been

Journal

JAMA Internal Medicine – American Medical Association

Published: Mar 1, 2019

References

  • Effect of mobile device–supported single-patient multi-crossover trials on treatment of chronic musculoskeletal pain: a randomized clinical trial
    Kravitz, RL; Schmid, CH; Marois, M
  • A randomized clinical trial of n-of-1 trials: tribulations of a trial
    Mirza, RD; Guyatt, GH