To investigate long-term (12-month) safety and symptom control of extended-release methylphenidate (MPH-MLR) in children aged 4 to < 6 years after treatment optimization.
Ninety children aged 4 to < 6 years with attention-deficit/hyperactivity disorder (ADHD) were enrolled from two MPH-MLR studies. Treatment-emergent adverse events (TEAEs) and ADHD symptom control were assessed in the safety population (n=89) and modeled with mixed model analyses.
Most TEAEs (89.9%) were rated by investigators as mild or moderate severity. One serious AE was reported (unrelated to study drug). Ten children discontinued due to TEAEs. Two discontinued due to weight loss; no significant increase in the rate of underweight children from baseline to endpoint was observed. Overall, 18% lost weight and 18% reported decreased appetite. Weight and height z-scores and obesity rates decreased significantly from baseline to endpoint. Insomnia was reported (9%), none of these children discontinued. Sleep quality did not change significantly. Hypertension was reported (6.7%); none of these children dropped out. Diastolic, but not systolic, blood pressure increased significantly during the follow-up. Control of ADHD symptoms was maintained throughout follow-up.
These data contribute to the understanding of the long-term safety of an extended-release stimulant in children 4 to < 6 years of age. The observed risk of a TEAE-related discontinuation was ∼11%. TEAEs were not dose-related and most were of mild to moderate severity. Symptom control was maintained through the year-long study.
A 12-Month Open Label Safety Study of Aptensio XR® in Children Ages 4-5 Years Diagnosed With ADHD (EF004);; NCT02677519.

Copyright © 2021. Published by Elsevier Inc.