Randomized Clinical Trials in Single Patients During a 2-Year Period

Larson, Eric B.; Ellsworth, Allan; Oas, Janet

doi:10.1001/jama.1993.03510220064035

Cited by 77 publications

(40 citation statements)

References 8 publications

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“…After emerging in the clinical literature to much fanfare [1,2,18], N-of-1 trials appeared in the literature sporadically during the late 1990s and then more consistently through the first decade of the current century. The apparent acceleration starting in 2010 may reflect a nexus between increased interest in personalized medicine and methods to enhance the delivery of patient-centered care [19].…”

Section: Discussionmentioning

confidence: 99%

Reporting quality of N-of-1 trials published between 1985 and 2013: a systematic review

Jiang

Gao

Punja

et al. 2016

Journal of Clinical Epidemiology

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Objective:To evaluate the quality of reporting of single-patient (N-of-1) trials published in the medical literature based on the CONSORT Extension for N-of-1 Trials (CENT) statement and to examine factors that influence reporting quality in these trials.Methods: Through a search of 10 electronic databases, we identified N-of-1 trials in clinical medicine published between 1 January 1985 and 31 December 2013. Two reviewers screened articles for eligibility and independently extracted data. Quality assessment was performed using the CENT statement. Discrepancies were resolved by consensus. Results:We identified 112 eligible N-of-1 trials published in 87 journals and involving a total of 2278 patients. Overall agreement between the two evaluators for compliance with CENT criteria was 0.80 (95% CI, 0.79 to 0.82). Trials assessed pharmacology and therapeutics (87%), behavior (11%), or diagnosis (2%). Although 87% of articles described the trial design (including the planned number of subjects and length of treatment period), the median percentage of specific CENT elements in the Methods was 41% (range 16% to 87%) and the median percentage in the Results was 38% (range 32% to 93%). First authors were predominantly from North America (46%), Europe (29%), and Australia (17%). Quality of reporting was higher in papers published in journals with relatively high impact factors (P=0.004). Conclusion:The quality of reporting of published N-of-1 trials is variable and needs improvement. Because the CENT guidelines were not published until near the end of the period of this review, these results represent a baseline from which improvement may be expected in the future.

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Section: Discussionmentioning

confidence: 99%

Reporting quality of N-of-1 trials published between 1985 and 2013: a systematic review

Jiang

Gao

Punja

et al. 2016

Journal of Clinical Epidemiology

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“…First, if the patient does not meet the eligibility criteria, extrapolation may not be appropriate; second, regardless of the overall trial results, some patients appear to benefit from experimental therapy and some do not. Therefore, to overcome the disadvantages of large-sample multicent er studies, the n-of-1 R CT has been developed in order to examine the intervention effect in individual patients using a corresponding methodology [11,12], This kind of single-patient trial has been validated by numerous stud ies [12,13,24], About two thirds of the conducted trials provided a definite clinical or statistical answer and gave a high level of confidence in therapy to the treating physi cian.…”

Section: Discussionmentioning

confidence: 99%

A Case Report of a Double-Blind, Randomized Trial of Inhaled Steroids in a Patient with Lung Transplant Bronchiolitis Obliterans

Speich

Boehler²,

Russi³

et al. 1997

Respiration

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Lung transplant bronchiolitis obliterans syndrome (BOS) is the most significant long-term cause of morbidity and mortality after lung transplantation. Although augmented immunosuppression is used by most centers, reported on treatment to reverse BOS are largely anecdotal. We performed a double-blind, randomized, controlled trial (RCT) with ten treatment pairs of 2 weeks duration each comparing inhaled fluticasone propionate (2 × 1,000 μg/day) with placebo in a patient with BOS grade 2 who previously showed an improvement in lung function after inhaled steroids. The Baseline Dyspnea Index and the Modified Medical Research Council Dyspnea Scale showed a significant improvement during fluticasone treatment compared with the placebo period (2.7 ± 0.2 vs. 2.0 ± 0.3; p = 0.043; and 1.7 ± 0.2 vs. 2.4 ± 0.2; p = 0.043). The patient correctly identified fluticasone and placebo, respectively, in eight of ten trial pairs (p = 0.016). The values of forced expiratory volume in 1 s were significantly higher during the fluticasone period (1,207 ± 10 ml; 95% confidence interval, CI, 1,187-1,227 ml) compared to the placebo period (1,150 ± 6ml;95%CI 1,138-1,162 ml; p = 0.0012). In conclusion, this n-of-1 RCT suggests the efficacy of high-dose inhaled fluticasone in our patient with lung transplant BOS. We propose to conduct a multicenter RCT of high-dose inhaled steroids. Until further data are available, this treatment modality should be offered to patients with lung transplant BOS.

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“…In most instances, treatments are applied to patients who don't meet criteria for inclusion in a randomized controlled trial. One method a clinician can consider for improving therapeutic precision is an n -of-1 clinical trial, 11,12 particularly when efficacy is truly in doubt and when treatment, if effective, will be continued long term. In this situation, the clinician solves the question of incorporating a research advance or treatment of uncertain value by performing effectiveness research on a single patient, using techniques developed for rigorous assessment of efficacy in cohorts of patients.…”

Section: Treating the Individual Patientmentioning

confidence: 99%