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/m² by 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/m² at 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.