Peer-reviewed article
Growth hormone treatment in children with Prader–Willi syndrome: safety and effectiveness data from the PATRO Children study
Constanze Lämmer, Philippe Ferdinand Backeljauw, Maïté Tauber, Shankar Kanumakala, Sandro Loche et al. ·
Summary
Written by AI, not yet AICS's view · claude-haiku-4-5-20251001This peer-reviewed study reports on the safety and effectiveness of growth hormone treatment in children with Prader-Willi syndrome, a rare genetic disorder. The research followed 235 children receiving the treatment over several years and monitored for side effects and growth outcomes. While the treatment was generally well tolerated and improved height, some serious side effects were observed, particularly sleep apnea and respiratory issues. The study concludes that close monitoring for known safety risks is important during treatment.
Key points
- 235 children with Prader-Willi syndrome enrolled; 86.4% were treatment-naïve at baseline
- Adverse events reported in 81.7% of patients; serious adverse events in 40.9%
- Most frequent treatment-related serious adverse events were sleep apnea (4.7%), tonsillar hypertrophy (1.7%), and adenoidal hypertrophy (1.7%)
- Mean height standard deviation score improved by +1.50 SD after 3 years of treatment
- Metabolic complications (impaired glucose tolerance, type 2 diabetes) were rare but occurred in individual cases
Limitations
This was a non-interventional postmarketing surveillance study without a control group, so it cannot definitively establish causation for adverse events or compare effectiveness to untreated children. The study reflects real-world clinical practice but lacks the rigorous design needed to isolate treatment effects from other factors.
Abstract
Background: Recombinant human growth hormone (rhGH, somatropin) therapy is approved in children with Prader–Willi syndrome (PWS). Objectives: To report safety and effectiveness data for children with PWS treated with biosimilar rhGH (Omnitrope ® , Sandoz) in the PAtients TReated with Omnitrope (PATRO) Children study. Design: PATRO Children was a multicenter, non-interventional, postmarketing surveillance study. Methods: Children with PWS received Omnitrope according to standard clinical practice. Adverse events (AEs) were monitored for the duration of Omnitrope treatment. Effectiveness outcomes were also assessed, including height standard deviation (SD) scores (HSDS). Results: As of July 2020 (study completion), 235 patients with PWS had been enrolled. At baseline, 95.7% ( n = 225) of patients were prepubertal and 86.4% ( n = 203) were rhGH treatment-naïve. At analysis, the median (range) treatment duration in the study was 56.8 (2.9–155.8) months. AEs were reported in 192 patients (81.7%) and were suspected as treatment-related in 39 patients (16.6%). Serious AEs (SAEs) were reported in 96 patients (40.9%) and were suspected as treatment-related in 22 patients (9.4%). The most frequent treatment-related SAEs were sleep apnea syndrome ( n = 11; 4.7%), tonsillar hypertrophy ( n = 4; 1.7%), and adenoidal hypertrophy ( n = 4; 1.7%). Development of scoliosis was considered treatment-related in two patients; development of impaired glucose tolerance in one patient and type 2 diabetes mellitus in another patient were considered treatment-related. Effectiveness outcomes were primarily assessed in 153 patients who completed 3 years of treatment. Among the 151 prepubertal patients (135 treatment-naïve), mean (SD) change from baseline in HSDS after 3 years was +1.50 (1.07) across all patients and +1.57 (1.07) for treatment-naïve patients. Conclusion: These data suggest that biosimilar rhGH is well tolerated and effective in patients with PWS managed in real-life clinical practice. Patients with PWS should continue to be closely monitored for well-known safety issues (including respiratory, sleep, and glucose metabolism disorders, and scoliosis) during rhGH treatment.