The efficacy and safety of weekly somatrogon treatment in turkish children with growth hormone deficiency: a real-world cohort study.

BACKGROUND

Long-acting growth hormone preparations have the potential to improve treatment compliance by reducing the frequency of injections. The aim of this study was to evaluate the efficacy and safety of weekly somatrogon treatment in patients with pediatric growth hormone deficiency (GHD) under real-world conditions.

METHODS

Patients with GHD aged 3-18 years who had been receiving somatrogon therapy for at least 6 months were included in the study. Anthropometric measurements, insulin-like growth factor-1 (IGF-1), bone age/chronological age (BA/CA) ratio, and drug-related adverse events were evaluated at baseline and at the final follow-up.

RESULTS

A total of 39 patients with a mean age of 12.90 ± 1.63 years were included. The mean treatment duration was 11.31 ± 2.72 months (min-max: 6.36-16.92). The mean annual height gain was 10.07 ± 0.61 cm, and the Δ height SDS was 0.64. No significant change was observed in the BA/CA ratio (p = 0.2). The mean baseline IGF-1 SDS was - 0.61 ± 1.15, increasing to 0.75 ± 1.26 at the final follow-up (p < 0.01). In 5 of 39 patients (12.8%), IGF-1 SDS exceeded + 2 SDS, requiring dose reduction. No serious adverse events were observed.

CONCLUSION

Based on our real-world data, weekly somatrogon therapy provided an effective growth response and was well tolerated in pediatric patients with GHD. In most cases, mean IGF-1 levels remained within the normal range. However, IGF-1 levels may be elevated, particularly in pubertal patients. These findings support somatrogon as a safe and effective early treatment option in routine clinical practice, although larger and longer-term studies are required to confirm long-term safety.