An n-of-1 trial service in clinical practice: Testing the effectiveness of stimulants for attention-deficit/hyperactivity disorder

Nikles, C. J., Mitchell, G. K., Del Mar, C. B., Clavarino, A. and McNairn, N. (2006) An n-of-1 trial service in clinical practice: Testing the effectiveness of stimulants for attention-deficit/hyperactivity disorder. Pediatrics, 117 6: 2040-2046.


Author Nikles, C. J.
Mitchell, G. K.
Del Mar, C. B.
Clavarino, A.
McNairn, N.
Title An n-of-1 trial service in clinical practice: Testing the effectiveness of stimulants for attention-deficit/hyperactivity disorder
Journal name Pediatrics  (ERA 2012 Listed)    (ERA 2010 Rank A)   Check publisher's open access policy
Publication date 2006
Sub-type Article
DOI 10.1542/peds.2005-1328
Volume number 117
Issue number 6
ISSN 2040-2046
Start page 2040
End page 2046
Total pages 7
Editor Lucey, J. F.
Place of publication Illinois, USA
Publisher American Academy of Pediatrics
Collection year 2006
Language eng
Subject C1
321210 Community Child Health
730204 Child health
Abstract OBJECTIVE. We sought to describe the clinical use of n-of-1 trials for attention-deficit/hyperactivity disorder in publicly and privately funded family and specialized pediatric practice in Australia. METHODS. We used a within-patient randomized, double-blind, crossover comparison of stimulant (dexamphetamine or methylphenidate) versus placebo or alternative stimulant using 3 pairs of treatment periods. Trials were conducted from a central location using mail and telephone communication, with local supervision by the patients' clinicians. PATIENTS. Our study population included children with clinically diagnosed attention-deficit/ hyperactivity disorder who were aged 5 to 16 years and previously stabilized on an optimal dose of stimulant. They were selected because treatment effectiveness was uncertain. MAIN OUTCOME MEASURES. Our measures included number of patients recruited, number of doctors who used the service, geographic spread, completion rates, response rate, and post-n-of-1 trial decisions. RESULTS. Forty-five doctors across Australia requested 108 n-of-1 trials, of which 86 were completed. In 69 drug-versus-placebo comparisons, 29 children responded better to stimulant than placebo. Immediately posttrial, 19 of 25 drug-versus-placebo responders stayed on the same stimulant, and 13 of 24 nonresponders ceased or switched stimulants. In 40 of 63 for which data were available, posttrial management was consistent with the trial results. For all types of n-of-1 trials, management changed for 28 of 64 children for whom information was available. DISCUSSION. Attention-deficit/hyperactivity disorder n-of-1 trials can be implemented successfully by mail and telephone communication. This type of trial can be valuable in clarifying treatment effect when it is uncertain, and in this series, they had a noticeable impact on short-term management.
Keyword Pediatrics
N-of-1 Trials
Attention-deficit/hyperactivity Disorder
Deficit Hyperactivity Disorder
Methylphenidate
Children
Medication
Adhd
Management
Diagnosis
Validity
Series
Q-Index Code C1

 
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