MON-035 - Once-Weekly Somatrogon in Pediatric Growth Hormone Deficiency: Real-World Efficacy, Safety, and Quality-of-Life Findings

Objective: To evaluate real-world 6- and 12-month clinical outcomes in children with growth hormone deficiency (GHD) treated with once-weekly somatrogon or daily somatropin, including patients who transitioned from somatropin to somatrogon.

Methods: Eligible patients were classified into three groups: somatrogon-naïve (naïve), somatrogon-switch (switch), and somatropin. All patients were followed for 6 or 12 months with standardized clinical and laboratory assessments. In the naïve and switch groups, body composition was assessed using bioimpedance analysis at baseline and at month 6. Standardized, age-appropriate assessments of overall quality of life were conducted using the Pediatric Quality of Life Inventory (PedsQL), the Child Behavior Checklist (CBCL), and the Multidimensional Scale of Perceived Social Support (MSPSS). Psychiatric evaluations were performed according to the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition, Text Revision (DSM-5-TR) criteria.

Results: A total of 58 patients (58.6% male) were included (naïve: n=20; switch: n=18; somatropin: n=20). Mean ages were 11.1±3.0, 9.7±3.4, and 10.5±3.2 years, respectively. After 12 months, mean changes in height SDS (Δheight SDS) were 0.6±0.3, 0.7±0.3, and 0.7±0.4; and height velocities were 10.0±1.9, 9.1±1.7, and 9.8±1.9 cm/year, respectively. Corresponding increases in IGF-1 SDS (ΔIGF-1 SDS) were 2.2±1.2, 0.9±1.2, and 1.3±1.0, respectively. Among the 38 patients receiving somatrogon, 15.8% (n=6; 3 naïve, 3 switch) developed IGF-1 SDS >+2 during follow-up, managed successfully with observation or dose adjustment. No serious adverse events were observed. Bioimpedance analyses demonstrated a favorable but non-significant trend toward improved body composition in somatrogon-naïve children. At six months, PedsQL domains, CBCL scales, and MSPSS scores remained stable (all p > 0.05).

Conclusion: Once-weekly somatrogon demonstrated efficacy and safety comparable to daily somatropin with stable quality of life and psychosocial outcomes in children with GHD.

*Unless otherwise noted, all abstracts presented at ENDO must not be released to the press or the public until the date and time of presentation. For oral presentations, the abstracts are embargoed until the session begins. The Endocrine Society reserves the right to lift the embargo on specific abstracts that are selected for promotion prior to or during ENDO.*