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

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.

The effects of body position on distortion-product Otoacoustic emission testing

Otoacoustic emissions are frequently acquired from patients in a variety of body positions aside from the standard, seated orientation. Yet little knowledge is available regarding whether these deviations will produce nonpathological changes to the clinical results obtained. The present study aimed to describe the effects of body position on the distortion-product otoacoustic emissions of 60 normal-hearing adults. With particular attention given to common clinical practice, the Otodynamics ILO292, and the measurement parameters of amplitude, signal-to-noise ratio, and noise were utilized. Significant position-related effects and interactions were revealed for all parameters. Specifically, stronger emissions in the mid frequencies and higher noise levels at the extreme low and high frequencies were produced by testing subjects while lying on their side compared with the seated position. Further analysis of body position effects on emissions is warranted, in order to determine the need for clinical application of position-dependent normative data.