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

To report real-world 6- and 12-month outcomes in children with growth hormone deficiency (GHD) treated with somatrogon or somatropin, including those who transitioned from somatropin to somatrogon.

Eligible patients were categorized into three groups [somatrogon-naïve (naïve), somatrogon-switch (switch), and somatropin only] and were followed for 6 or 12 months. Bioimpedance analysis, as well as a standardized, age-appropriate assessment of the Pediatric Quality of Life Inventory (PedsQL), the Child Behavioural Checklist (CBCL) and the Multidimensional Scale of Perceived Social Support (MSPSS), were conducted at baseline and month 6 in the naïve and switch groups. Psychiatric evaluations were also performed according to the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition, Text Revision (DSM-5-TR) criteria.

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 standard deviation score (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).

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