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Safety and efficacy of weekly somapacitan for growth hormone deficiency in children: A 72-week observational study
Abdulmoein E Al-Agha1, Hasan M Aljefri1, Shahd E Kafi1
1From the Department of Pediatric (Al-Agha, Aljefri, Kafi, Alamri); and from the Department of Community Medicine (Alghamdi), King Abdulaziz University Hospital, Jeddah, Kingdom of Saudi Arabia.
Insights
Weekly somapacitan effectively treats pediatric growth hormone deficiency (GHD), improving height velocity and insulin-like growth factor 1 (IGF-1) levels. This long-acting growth hormone analog demonstrates good safety and tolerability in children with GHD.
Area of Science:
- Pediatric Endocrinology
- Growth Hormone Therapy
- Metabolic Disorders
Background:
- Pediatric growth hormone deficiency (GHD) requires effective and adherence-friendly treatment options.
- Long-acting growth hormone analogs offer potential improvements in treatment adherence compared to daily injections.
- Somapacitan is a novel, once-weekly growth hormone analog for managing pediatric GHD.
Purpose of the Study:
- To evaluate the efficacy and safety of weekly somapacitan in pediatric GHD patients.
- To assess the impact of somapacitan on height velocity and insulin-like growth factor 1 (IGF-1) levels.
- To determine the treatment adherence and tolerability of somapacitan in a real-world clinical setting.
Main Methods:
- An observational study involving 184 children with GHD was conducted over 72 weeks.
- Patients received weekly somapacitan doses, adjusted based on IGF-1 levels.
- Key outcome measures included changes in height velocity and IGF-1 levels, with statistical significance at p < 0.05.
Main Results:
- Significant improvements in IGF-1 levels (from 166.97 to 363.74) and height velocity were observed by week 72.
- Height velocity gains were most pronounced in children at Tanner Stage 1; treatment-naive children showed greater initial response.
- Somapacitan was well-tolerated with minimal adverse events, indicating a favorable safety profile for long-term use.
Conclusions:
- Weekly somapacitan is an effective and safe treatment for pediatric GHD, allowing for individualized dosing via IGF-1 monitoring.
- Routine IGF-1 monitoring is recommended to optimize treatment benefits and minimize potential risks.
- Further research in diverse pediatric populations is warranted to confirm these findings in pediatric endocrinology.
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.
