EVIDENCE-BASED ANSWER
Stimulant medication therapy is the most effective treatment for attention deficit/hyperactivity disorder (ADHD) in children, producing significant improvements in symptoms and modest improvements in academic achievement (strength of recommendation [SOR]: A, based on multiple randomized controlled trials [RCTs]). Nonpharmacologic therapies, such as behavior therapy, school-based interventions, and family therapy, are not as effective as stimulants but may add modest benefit to the effects of medication (SOR: B, based on 1 RCT).
While atomoxetine (Strattera) improves the symptoms of ADHD (SOR: A, based on multiple RCTs), stimulant medications other than methylphenidate offer no distinct short-term advantages (SOR: A, based on meta-analyses of multiple RCTs). Combination drug therapies offer no significant advantage to stimulants alone unless a comorbid condition is present (SOR: A, based on a meta-analysis of 20 RCTs).
The combination of methylphenidate and clonidine (Catapres) improves symptoms in children with both ADHD and tics (SOR: B, based on 1 RCT). Clonidine is less effective alone and has significant side effects (SOR: B, based on a metaanalysis of nonrandomized trials).
Evidence summary
In numerous systematic reviews, RCTs, and metaanalyses, 70% of children responded to stimulant medications with short-term improvements in ADHD symptoms (inattention and hyperactivity/ impulsivity) and academic achievement. A fortyyear review looked at 135 trials and 413 RCTs of methylphenidate in over 19,000 children with an average age of 8.8 years (range, 8.3–9.4 years) for an average duration of 6 weeks (range, 3.3–8.0 weeks).1-3
Study groups included mostly elementary school–aged male children, with few minorities represented. Comorbid conditions, present in 65% of children with ADHD, were often poorly controlled. Outcome measures varied among studies.3
The effect size from stimulant medication in these studies averaged 0.8 for symptom relief and between 0.4 and 0.5 for academic achievement. (Effect size is the difference between the means of the experimental and control groups expressed in standard deviations. An effect size of 0.2 is considered small, 0.5 is medium, and 0.8 is considered moderate to large.)
A large randomized trial of 579 children with ADHD (20% girls) aged 7 to 9.9 years compared outcomes of 4 treatment strategies: stimulant medication, intensive behavioral treatment, combined stimulant medication and behavioral interventions, and standard community care.4 All children met the DSM-IV. criteria for ADHD Combined Type (the most common type of ADHD in this age group). The stimulant medication strategy included an initial dose titration period followed by monthly 30-minute visits. Intensive behavioral treatment involved child, parent, and school personnel components of therapy. Combination therapy added the regimens for medication and behavioral treatment together. Standard community care consisted of usual (nonsystematic) care, evaluated at 6 different sites.
After 14 months of treatment, children in the medication group and the combined treatment groups showed more improvement in ADHD symptoms than children given intensive behavioral treatment or those who received standard community care. When combined with medication, those treated with behavioral therapy showed slight improvement in social skills, anxiety, aggression, oppositional behavior, and academic achievement over medication alone. At the conclusion of the study, 74% of the 212 children on medication were successfully maintained on methylphenidate alone, 10% required dextroamphetamine, and no children required more than one medication. This study found that higher doses of medication with more frequent office follow-up and regular school contact were important features of successful treatment. Only 40% of families were able to complete the intensive behavioral therapy.
Several short-term reviews and meta-analyses show that side effects from stimulant medications are mild and have short duration.5 More long-term studies are required to evaluate effects on growth. RCTs have limited power to detect rare adverse events that may be better detected by large observational studies.6