ONCE-WEEKLY SOMATROGON IN PEDIATRIC GROWTH HORMONE DEFICIENCY: REAL-WORLD EFFICACY, SAFETY, AND QUALITY-OF-LIFE FINDINGS

Kübra ŞEN KÜÇÜK, Sebla GÜNEŞ, Mustafa DİNÇER, Sercan ÖZTÜRK, Tolga ÜNÜVAR, Ahmet ANIK

Journal of Clinical Research in Pediatric Endocrinology - 2026;18(2):323-332

Aydın Adnan Menderes University Faculty of Medicine, Department of Pediatrics, Division of Pediatric Endocrinology, Aydın, Türkiye

 

Objective: 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. Methods: 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. 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 standard deviation score (Deltaheight 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 (DeltaIGF-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.