Double-blind methylphenidate trials - Practical, useful, and highly endorsed by families

Objective: To evaluate a 3-week, randomized, double-blind, methylphenidate placebo-controlled trial (MPT) in routine practice for children with attent ion-deficit disorder. Patients and Methods: School-aged children with attention-deficit/hyperacti vity disorder (Diagnostic and Statistical Manual of Mental Disorders, Fourt h Edition criteria) who enrolled an "N of 1" trial at a pediatric tertiary care center were eligible. Families (n = 50) with a child eligible for the MPT were given 3 bottles of identical capsules. The capsules contained, in random order: placebo of the prescribed dose of methylphenidate (Ritalin) h ydrochloride (0.3 mg/kg or 0.6 mg/kg). Families gave the child 1 capsule at 8 AM and 1 capsule at noon. The family, teacher, and physician were blinde d for the order of medication. Conners questionnaires (Conners Parent Quest ionnaire and Conners Teacher Questionnaire) and written comments were compl eted by parents and teachers at baseline and at the end of each week. Once MPT results were known and following discussion with the physician, familie s decided whether to continue methylphenidate therapy. Families were interv iewed by telephone 14 to 21 months after the MPT. Results: Forty-three (86%) of the 50 eligible children (mean age, 129 month s) were contacted. No family found the MPT difficult, but 6 trials were inc omplete, usually because of side effects. All families used the MPT to deci de if methylphenidate was the correct treatment choice for their child and 68% (34 of 50 families) used the results exclusively. The remaining 16 fami lies believed the MPT was helpful. Overall, 31 (72%) of the 43 children had a good response to methylphenidate treatment-20 (47%) continued to use it for longer than 12 months and 8 (26%) for 2 to 12 months; 3 responders chos e not to use it after the MPT. Nine of the 43 families chose not to use met hylphenidate treatment; however, all indicated that participating in the MP T helped them to make that decision. In follow-up interviews, the same prop ortion of methylphenidate users and nonusers reported improvement in many a reas of function including significantly less time spent doing homework. Us ers reported reduced aggression (P<.001) and fewer discipline problems (P<. 01) compared with nonusers. Conclusions: An "N of 1" MPT was easily performed and permitted families to decide whether to use methylphenidate for long-term treatment of attention -deficit disorder or attention-deficit/hyperactivity disorder. Regardless o f methylphenidate use or lack of use, the condition of all of these childre n was improved at follow-up.