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Growth hormone treatment in children with Prader-Willi syndrome: safety and effectiveness data from the PATRO

Constanze Lämmer1, Philippe Backeljauw2, Maite Tauber3

  • 1Department of Pediatrics, KJF Josefinum, Joseph-Mayer-Straße 1, Augsburg 86154, Germany.

Therapeutic Advances in Endocrinology and Metabolism
|October 7, 2024
PubMed

Insights

Biosimilar recombinant human growth hormone (rhGH) is safe and effective for children with Prader-Willi syndrome (PWS). The study monitored adverse events and found positive height standard deviation score changes after three years of treatment.

Area of Science:

  • Pediatric Endocrinology
  • Genetics and Inherited Diseases

Background:

  • Prader-Willi syndrome (PWS) is a genetic disorder affecting multiple body systems.
  • Recombinant human growth hormone (rhGH) therapy is an approved treatment for children with PWS.

Purpose of the Study:

  • To evaluate the safety and effectiveness of biosimilar rhGH (Omnitrope®) in children with PWS.
  • To collect real-world data on Omnitrope treatment in a pediatric PWS population.

Main Methods:

  • A multicenter, non-interventional, postmarketing surveillance study (PATRO Children).
  • 235 children with PWS received Omnitrope per standard clinical practice.
  • Adverse events (AEs) and effectiveness outcomes, including height standard deviation scores (HSDS), were monitored.

Main Results:

  • Over a median of 56.8 months, 81.7% of patients experienced AEs, with 16.6% suspected as treatment-related.
  • Serious AEs (SAEs) occurred in 40.9% of patients, with 9.4% suspected as treatment-related. Most frequent treatment-related SAEs included sleep apnea syndrome.
  • After 3 years, treatment-naïve prepubertal patients showed a mean HSDS increase of +1.57 (SD 1.07).

Conclusions:

  • Biosimilar rhGH (Omnitrope®) demonstrates good tolerability and effectiveness in children with PWS in real-world settings.
  • Continued monitoring for known safety issues (respiratory, sleep, glucose metabolism, scoliosis) is recommended during rhGH therapy for PWS.
Abstract

Keywords:
Omnitrope®PATRO ChildrenPrader–Willi syndromebiosimilargrowth hormone replacement therapysomatropin

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