PATRO children, a multi-center, non-interventional study of the safety and effectiveness of Omnitrope®(somatropin) treatment in children: Update on the United States cohort

Philippe Backeljauw, Bradley S. Miller, Richard Levy, Kenneth McCormick, Hichem Zouater, Markus Zabransky, Kim Campbell

Research output: Contribution to journalArticlepeer-review

Abstract

Objectives: Omnitrope® (somatropin, Sandoz Inc.) is one of several recombinant human growth hormones (rhGH) approved in the United States (US) for use in pediatric indications, including growth hormone deficiency (GHD) and idiopathic short stature (ISS). We report data on the effectiveness and safety of Omnitrope® in the US cohort of the PATRO Children (international, longitudinal, non-interventional) study. Methods: All visits and assessments are carried out according to routine clinical practice, and doses of Omnitrope® are given according to country-specific prescribing information. Results: By September 2018, 294 US patients were recruited; the two largest groups were GHD (n=193) and ISS (n=62). Across all indications, HSDS improvement (ΔHSDS) from baseline at three years was +1.0 (rhGH-naïve, +1.2; pre-treated, +0.7). In pre-pubertal patients, ΔHSDS from baseline at three years was +0.94 (rhGH-naïve, +1.3; pre-treated, +0.7). Following three years of treatment, ΔHSDS from baseline was +1.3 in rhGH-naïve GHD patients and +1.1 in rhGH-naïve ISS patients. In pre-pubertal rhGH-naïve patients, ΔHSDS from baseline was +1.3 and +1.2 in GHD and ISS patients, respectively. Overall, 194 patients (66.0%) experienced adverse events (AEs; n=886 events); most were of mild-moderate intensity. Five patients (1.7%) had AEs that were suspected to be treatment-related (n=5 events). All reported neoplasms were benign, non-serious, and considered unrelated to rhGH therapy. No AEs of diabetes mellitus or hyperglycemia were reported. Conclusions: Omnitrope® appears to be well tolerated and effective in the majority of patients, without evidence of an increased risk of developing unexpected AEs, diabetes mellitus, or new malignancies during treatment.

Original languageEnglish (US)
Pages (from-to)431-440
Number of pages10
JournalJournal of Pediatric Endocrinology and Metabolism
Volume34
Issue number4
DOIs
StatePublished - Apr 1 2021

Bibliographical note

Funding Information:
Research funding: The PATRO Children study is funded by Sandoz Biopharmaceuticals, c/o HEXAL AG.

Funding Information:
The authors thank Yu-Ming Shen (Sandoz/Hexal AG) for providing statistical support. Medical writing support was provided by Tony Reardon of Spirit Medical Communications Ltd, supported by Sandoz/Hexal AG.

Publisher Copyright:
© 2020 2020 Philippe Backeljauw et al., published by De Gruyter, Berlin/Boston.

Keywords

  • Omnitrope
  • growth hormone
  • growth hormone deficiency
  • idiopathic short stature
  • pediatrics

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