Safety and Effectiveness of Recombinant Human Growth Hormone in Children with Turner Syndrome: Data from the PATRO
Philippe Backeljauw1, Shankar Kanumakala2, Sandro Loche3
1Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
Insights
Biosimilar recombinant human growth hormone (rhGH) is safe and effective for treating Turner syndrome (TS) in children. Real-world data show positive height outcomes, suggesting rhGH is a valuable treatment option for TS patients.
Area of Science:
- Pediatrics
- Endocrinology
- Pharmacovigilance
Background:
- PATRO Children is an international, observational, postmarketing surveillance study of biosimilar recombinant human growth hormone (rhGH; somatropin, Omnitrope®).
- This study reports safety and effectiveness data specifically for patients diagnosed with Turner syndrome (TS).
Purpose of the Study:
- To evaluate the safety and effectiveness of biosimilar rhGH (Omnitrope®) in pediatric patients with Turner syndrome (TS).
- To assess real-world outcomes, including adverse events and height development, in TS patients receiving rhGH treatment.
Main Methods:
- The study enrolled infants, children, and adolescents with TS receiving Omnitrope® under standard clinical practice.
- Safety was monitored through adverse event (AE) reporting. Effectiveness was evaluated by calculating height velocity (HV), height standard deviation score (HSDS), and HVSDS.
Main Results:
- 348 TS patients were enrolled; 90.2% were prepubertal and 81.6% were rhGH treatment-naïve at baseline.
- 48.9% of patients reported AEs, with 7.2% considered treatment-related; one serious treatment-related AE (intracranial hypertension) occurred.
- After 3 years, treatment-naïve prepubertal patients showed a mean ΔHSDS of +1.17. Patients reaching adult height (AH) achieved a mean AHSDS of -2.02.
Conclusions:
- Biosimilar rhGH (Omnitrope®) demonstrates good tolerability and effectiveness in Turner syndrome patients within real-life clinical settings.
- The findings suggest that rhGH is a viable treatment option for improving growth outcomes in TS.
- Optimizing rhGH dosage may further enhance the achievement of adult height in TS patients.
Introduction:
PATRO Children is an international, observational, postmarketing surveillance study for a biosimilar recombinant human growth hormone (rhGH; somatropin, Omnitrope®; Sandoz), approved by the European Medicines Agency in 2006. We report safety and effectiveness data for patients with Turner syndrome (TS).
Methods:
The study population included infants, children, and adolescents with TS who received Omnitrope® treatment according to standard clinical practice. Adverse events (AEs) were monitored for safety evaluation, and height velocity (HV), height standard deviation score (HSDS), and HVSDS were calculated to evaluate treatment effectiveness.
Results:
As of August 2019, 348 TS patients were enrolled from 130 centers. At baseline, 314 patients (90.2%) were prepubertal and 284 patients (81.6%) were rhGH treatment naïve. The mean (range) age at baseline was 9.0 (0.7-18.5) years, and mean (SD) treatment duration in the study was 38.5 (26.8) months. Overall, 170 patients (48.9%) reported AEs, which were considered treatment related in 25 patients (7.2%). One treatment-related serious AE was reported (intracranial hypertension). Mean ΔHSDS after 3 years of therapy was +1.17 in treatment-naïve prepubertal patients and +0.1 in pretreated prepubertal patients. In total, 51 patients (31.1%) reached adult height (AH), 35 of whom were rhGH treatment naïve; in these patients, mean (SD) HSDS was -2.97 (1.03) at the start of Omnitrope® treatment, and they achieved a mean (SD) AHSDS of -2.02 (0.9).
Conclusion:
These data suggest that biosimilar rhGH is well tolerated and effective in TS patients managed in real-life clinical practice. Optimization of rhGH dose may contribute to a higher AH.
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