Abstract
OBJECTIVES: To assess the effectiveness and safety of somapacitan, a long-acting growth hormone analog, as a promising treatment to improve adherence in pediatric growth hormone deficiency (GHD) management. METHODS: The observational study included 184 children diagnosed with GHD. Carried out over 72 weeks at the Pediatric Endocrinology Outpatient Clinic in collaboration with King Abdulaziz University Hospital in Jeddah, Saudi Arabia. Patients received weekly somapacitan doses, starting at 0.16 mg/kg and adjusted according to insulin-like growth factor 1 (IGF-1) levels. Key outcomes included changes in height velocity and IGF-1 levels, with statistical significance set at a p-value of <0.05. RESULTS: The cohort, with an average age of 10.1 years and 67.4% male, showed substantial improvements by week 72: IGF-1 levels increased from 166.97 to 363.74, and height velocity gains were most pronounced in children at Tanner Stage 1. Treatment-naive children exhibited greater initial height velocity improvements compared to those switching from daily GH therapy, although these differences leveled over time. Somapacitan was well-tolerated, with minimal side effects, supporting its long-term safety. CONCLUSION: Weekly somapacitan offers an effective, safe pediatric GHD treatment, with individualized dosing based on IGF-1 levels. Routine IGF-1 monitoring is advised to maximize benefits and minimize risks. Further studies on diverse populations are recommended to validate these findings in pediatric endocrinology.