Using a Patient-Centered Outcome Measure to Test Methylphenidate Versus Placebo in Children with Autism Spectrum Disorder

Abstract Objectives: Parent rating scales are commonly used to evaluate change in clinical trials. Despite advantages, these measures may not capture parental impression of the child's most salient problems. We examine the use of parent target problems (PTPs) in a randomized trial of methylphenidate (MPH) in children with autism spectrum disorder and symptoms of attention-deficit/hyperactivity disorder. Methods: This multisite, 4-week, randomized crossover trial compared three dose levels (low, medium, and high) of MPH with placebo. At baseline, the independent evaluator (IE) asked parents to nominate the child's two biggest problems. For each problem, the IE and parent coconstructed a brief narrative of the behavior and the impact on family life. The IE and parents reviewed and revised the narratives at subsequent visits. A panel of four judges, blind to treatment condition, independently reviewed the narratives to rate change from baseline on a 9-point scale: 1, normal; 2, markedly improved; 3, definite...