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Correspondence to Author: Pars Jävendah, 

Department of Women’s and Children’s Health, Karolinska Institutet and Pediatric Endocrinology Unit, Karolinska University Hospital, 171 64 Solna, Sweden.

ABSTRACT: 
Context : The daily injections required for current GH therapy can be taxing. A long-acting GH derivative called somapacitan is being developed to treat GH deficit (GHD).
Goal : After three years of treatment, assess the safety, effectiveness, and tolerability of once-weekly somapacitan.
Design : A multicenter, randomized, controlled, phase 2 research (NCT02616562) comparing once-daily GH with somapacitan lasted 156 weeks.
Location : 29 locations throughout 11 nations. Patients : A total of fifty-nine GHD children were randomly assigned (1:1:1:1) and given therapy. After three years, fiftythree youngsters were done.
Interventions : During the first year of treatment, patients received subcutaneous injections of somapacitan (0.04 [n = 14], 0.08 [n = 15], or 0.16 [n = 14] mg/kg/wk) or daily GH (n = 14) (0.034 mg/kg/d, or 0.238 mg/kg/wk). After that, all somapacitan patients received 0.16 mg/kg/wk.
Key Outcome Measures : Changes from baseline in height SD score (HSDS), height velocity (HV) at year three, and IGF-I SDS.
Findings : In comparison to daily GH, the estimated treatment difference (95% CI) in HV for somapacitan at year three was 0.8 cm/y (−0.4 to 2.1). The difference in HVSDS between the daily GH, the pooled somapacitan groups, and somapacitan 0.16/0.16 mg/kg/wk was similar from baseline to year 3. All groups showed a progressive rise in HSDS from baseline. The mean HSDS at year three was comparable for daily GH and the pooled somapacitan groups. Treatment differences in mean IGF-I SDS from baseline to year three were comparable.
Conclusions : In children with GHD, once-weekly somapacitan demonstrated maintained efficacy over three years in all evaluated height-based outcomes, while also being comparably safe and tolerable to daily GH. For this study, there is a simple language summary (1) accessible.

Citation:

Pars Jävendah. Comparing Daily GH with Once-Weekly Somapacitan for Effective GH Replacement in Children With GHD: Three-Year Results From REAL 3. Journal of Clinical Endocrinology and Metabolism 2024.