Family-Based Treatment of Early Childhood Obsessive-Compulsive Disorder The Pediatric Obsessive-Compulsive Disorder Treatment Study for Young Children (POTS Jr)-A Randomized Clinical Trial

IMPORTANCE Cognitive behavior therapy (CBT) has been established as efficacious for obsessive-compulsive disorder (OCD) among older children and adolescents, yet its effect on young children has not been evaluated sufficiently. OBJECTIVE To examine the relative efficacy of family-based CBT (FB-CBT) involving exposure plus response prevention vs an FB relaxation treatment (FB-RT) control condition for children 5 to 8 years of age. DESIGN, SETTING, AND PARTICIPANTS A 14-week randomized clinical trial (Pediatric Obsessive-Compulsive Disorder Treatment Study for Young Children [POTS Jr]) conducted at 3 academic medical centers between 2006 and 2011, involving 127 pediatric outpatients 5 to 8 years of age who received a primary diagnosis of OCD and a Children's Yale-Brown Obsessive Compulsive Scale total score of 16 or higher. INTERVENTIONS Participants were randomly assigned to 14 weeks of (1) FB-CBT, including exposure plus response prevention, or (2) FB-RT. MAIN OUTCOMES AND MEASURES Responder status defined as an independent evaluator-rated Clinical Global Impression-Improvement scale score of 1 (very much improved) or 2 (much improved) and change in independent evaluator-rated continuous Children's Yale-Brown Obsessive Compulsive Scale total score. RESULTS Family-based CBT was superior to FB-RT on both primary outcome measures. The percentages of children who were rated as 1 (very much improved) or 2 (much improved) on the Clinical Global Impression-Improvement scale at 14 weeks were 72% for FB-CBT and 41% for FB-RT. The effect size difference between FB-CBT and FB-RT on the Clinical Global Impression-Improvement scale was 0.31 (95% CI, 0.17-0.45). The number needed to treat (NNT) with FB-CBT vs FB-RT was estimated as 3.2 (95% CI, 2.2-5.8). The effect size difference between FB-CBT and FB-RT on the Children's Yale-Brown Obsessive Compulsive Scale at week 14 was 0.84 (95% CI, 0.62-1.06). CONCLUSIONS AND RELEVANCE A comprehensive FB-CBT program was superior to a relaxation program with a similar format in reducing OCD symptoms and functional impairment in young children (5-8 years of age) with OCD.