Attention-deficit hyperactivity disorder (ADHD) is a common comorbidity in Duchenne muscular dystrophy (DMD). Until now, treatment with methylphenidate (MPH) has never been systematically assessed and described in this population. Here, the authors aimed was to evaluate the effectiveness and safety of short acting MPH for learning problems in males with DMD and ADHD. Neuropsychological (cognition and behaviour) and medical data of a sample of ten males (mean age = 8.1 years, range 6.3-9.8) with DMD and an ADHD diagnosis was retrospectively analysed at baseline (T0; without MPH), short-term follow-up (T1; with MPH; mean interval T0-T1 = 8.3 months, range 4.3-15.6), and long-term follow-up (T2; mean interval T1-T2 = 23.1 months, range 2.6-77.7). An initial MPH dose of 5 mg/day was given on school mornings, with an increase of 2.5-5 mg/week depending on individual tolerance and treatment response, until a sufficiently effective dose was reached (range 0.2-0.6 mg/kg/day). At T1, results demonstrated an improvement in attention (i.e. concentration, impulsivity, and distractibility) in four patients. Suboptimal effects were reported in four patients, and no effects in two patients. At T2, seven patients showed considerable improvement in attention. No major side effects were reported. Overall, the data show that short acting MPH can be clinically effective for learning problems in males with DMD and ADHD, with regular cardiac follow-up, and close monitoring of side effects and neuropsychological effects.
