Abstract

Most major psychiatric disorders have an onset in childhood or adolescence in a sizeable proportion of patients, and earlier onset disorders often have a severe and chronic course that can seriously disrupt sensitive developmental periods, with lifelong adverse consequences. Accordingly, psychopharmacologic treatments have been increasingly utilized in severely ill youth. However, the increased use of psychopharmacologic treatments in pediatric patients has also raised concerns regarding a potential overdiagnosis and overtreatment of youth, without adequate data regarding the pediatric efficacy and safety of psychotropic agents. Over the past decade, a remarkable number of pediatric randomized controlled trials have been completed, especially with psychostimulants, antidepressants, and antipsychotics. For these frequently used agents, effect sizes against placebo have typically been at least moderate, with most numbers-needed-to-treat well below 10 for response, indicating clinical significance as well. Nevertheless, data also point to a greater and/or different profile of susceptibility to adverse effects in pediatric compared to adult patients, as well as to a role for nonpharmacologic treatments, given alone or combined with pharmacotherapy, for many of the youth. Taken together, these results highlight the need for a careful assessment of the risk-benefit relationship of psychopharmacologic treatments in patients who cannot be managed sufficiently with nonpharmacologic interventions and for routine, proactive adverse effect monitoring and management. Although considerable progress has been made, there is still enormous need for additional data and funding of pediatric psychopharmacology trials. It is hoped that the field will acquire the necessary resources to propel pediatric clinical psychopharmacology to new levels of insight by linking it with, but not replacing it by, pharmacoepidemiologic and biomarker approaches and advances.

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