Use of obesity medications in a young pediatric patient with optic nerve hypoplasia and severe early-onset obesity: A case report.

INTRODUCTION: Pediatric obesity presents complex challenges in children with underlying endocrine disorders. In patients with optic nerve hypoplasia (ONH) and hypothalamic-pituitary dysfunction, lifestyle changes alone are often ineffective for obesity treatment. This case report describes targeted pharmacologic interventions for severe early-onset obesity in a child with ONH and multiple pituitary hormone deficiencies. CASE PRESENTATION: A Hispanic female was diagnosed at 45 days of life with ONH and central arginine vasopressin deficiency, central hypothyroidism, secondary adrenal insufficiency, and growth hormone deficiency. Despite early interventions, her weight-for-length percentile rose from the 65th to >97th by nine months, with continued acceleration to body mass index (BMI) of 52 kg/mby age 5. Topiramate 100 mg nightly was initiated, with some associated appetite suppression and weight deceleration appreciated. Weight continued to increase during the COVID-19 pandemic, and at age 6, the dose of topiramate was increased (200 mg nightly) and phentermine 15 mg daily was added. An 11 % BMI reduction was observed over the subsequent 12 months, leading to BMI of 47 kg/mat age 7. Due to persistent and worsening obstructive sleep apnea (OSA) despite BMI reduction, semaglutide was then initiated, with ramp-up to 2.4 mg weekly, resulting in BMI reduction of 30 % over 24 months with triple-agent therapy, and associated resolution of OSA, elevated liver enzymes, and hypertriglyceridemia. DISCUSSION: This case highlights the potential role and therapeutic benefit of early, targeted pharmacologic intervention in managing severe obesity in pediatric patients with hypothalamic dysfunction and ONH. In a setting where lifestyle modifications alone are insufficient, the use of combination obesity medications resulted in substantial and sustained BMI reduction, alongside resolution of obesity-related comorbidities. These findings underscore the need for proactive, individualized treatment strategies in complex pediatric obesity.