Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2024 Mar 2:18:667-684.
doi: 10.2147/DDDT.S440009. eCollection 2024.

Safety and Effectiveness of a Biosimilar Recombinant Human Growth Hormone in Children Requiring Growth Hormone Treatment: Analysis of Final Data from PATRO Children, an International, Post-Marketing Surveillance Study

Sandro Loche  1 , Shankar Kanumakala  2 , Philippe Backeljauw  3 , Karl Otfried Schwab  4 , Alfonso M Lechuga-Sancho  5   6   7 , Altaher Esmael  8 , Dragan Urosevic  9 , Anca Boldea  8 , Markus Zabransky  8
Affiliations

Safety and Effectiveness of a Biosimilar Recombinant Human Growth Hormone in Children Requiring Growth Hormone Treatment: Analysis of Final Data from PATRO Children, an International, Post-Marketing Surveillance Study

Sandro Loche et al. Drug Des Devel Ther. .

Abstract

Purpose: Omnitrope® (somatropin) was approved as a biosimilar recombinant human growth hormone (rhGH) in 2006. Here, we report final data from the PAtients TReated with Omnitrope® (PATRO) Children study, a post-marketing surveillance study designed to monitor the long-term safety and effectiveness of this treatment in pediatric patients.

Methods: The study population included all pediatric patients treated with Omnitrope® (biosimilar rhGH), administered via daily injection, in routine clinical practice. The primary objective was to assess long-term safety, with effectiveness assessed as a secondary objective.

Results: In total, 7359 patients were enrolled and treated in the PATRO Children study; 86.0% were treatment-naïve at baseline. Growth hormone deficiency was the most frequent indication (57.9%), followed by patients born small for gestational age (SGA; 26.6%). The mean (SD) duration of exposure to biosimilar rhGH was 3.66 years (2.39). A total of 16,628 adverse events (AEs) were reported in 3981 (54.1%) patients, most of which were mild/moderate. AEs suspected to be treatment related occurred in 8.3% of patients, most frequently headache (1.6%), injection-site pain (1.1%), or injection-site hematoma (1.1%). The incidence rate (IR) of type 2 diabetes mellitus was 0.11 per 1000 person-years (PY) across all patients, and 0.13 per 1000 PY in patients born SGA. The IR of newly diagnosed primary malignancies was 0.22 per 1000 PY across all patients. In the 6589 patients included in the effectiveness population, a sustained catch-up growth was observed across all indications. After 5 years of treatment, height SDS increased from baseline by a median (range) of +1.79 (-3.7 to 6.2) in treatment-naïve patients and +0.73 (-1.4 to 3.7) in pretreated patients.

Conclusion: This final analysis of the PATRO Children study indicates that biosimilar rhGH is well tolerated and effective in real-world clinical practice. These data are consistent with the well-characterized safety profile of rhGH treatment in pediatric patients.

Keywords: Omnitrope; PATRO Children; growth hormone; growth hormone deficiency; pediatrics; small for gestational age.

PubMed Disclaimer

Conflict of interest statement

Sandro Loche is a member of the PATRO Children Advisory Board and the PATRO Children Global Steering Committee, and has received advisory board and lecture fees from Sandoz, Merck Serono, Ipsen, and Pfizer. Shankar Kanumakala has received support to attend scientific meetings from Sandoz in previous years, and is a member of the PATRO Children Global Steering Committee. Philippe Backeljauw is a member of the PATRO Children Advisory Board, and has received advisory board, consultancy, and lecture fees from Novartis/Sandoz, BioMarin, Ascendis, Tolmar, Cavalry Biosciences, Novo Nordisk, and Ipsen. Karl Otfried Schwab is a member of the German PATRO Children Advisory Board and the PATRO Children Advisory Board, and has received advisory board and lecture fees from Akcea, Alexion, Merck Healthcare, Novartis, Pfizer, and Sanofi-Aventis. Alfonso M Lechuga-Sancho is a member of an external advisory board for Sandoz and Ipsen, and has received research grants from Abbott, Medtronic, Menarini, Merck Serono, Sanofi, and Novo Nordisk. Altaher Esmael and Markus Zabransky are employees of HEXAL AG (a Sandoz company). Dragan Urosevic is an employee of Novartis Sandoz Biopharmaceutical AG. Anca Boldea was an employee of HEXAL AG (a Sandoz company) when the study and manuscript development were undertaken. The authors report no other conflicts of interest in this work.

Figures

Figure 1
Figure 1
Patient enrollment (%) by indication and country.
Figure 2
Figure 2
Percentage of patients at each visit with IGF-I values lower than, within, or above normal range (treatment-naïve patients in the safety analysis set with available data).
Figure 3
Figure 3
Yearly increase in height SDS in treatment-naïve patients, by indication. Figure shows box plots for the difference between height SDS in the year specified and height SDS 1 year before.
Figure 4
Figure 4
Yearly increase in height velocity SDS (PC) in treatment-naïve patients, by indication. Figure shows box plots for the difference between height velocity SDS in the year specified and height velocity SDS 1 year before.
Figure 5
Figure 5
Mean difference between projected height and targeted height by visit (effectiveness population).
See this image and copyright information in PMC

Similar articles

See all similar articles

Cited by

References

    1. European Medicines Agency [Internet]. Omnitrope® summary of product characteristics, updated 2023; 2008. Available from: https://www.ema.europa.eu/en/documents/product-information/omnitrope-epa.... Accessed June 30, 2023.
    1. Tidblad A. The history, physiology and treatment safety of growth hormone. Acta Paediatr. 2022;111(2):215–224. doi:10.1111/apa.15948 - DOI - PubMed
    1. Ranke MB, Wit JM. Growth hormone – past, present and future. Nat Rev Endocrinol. 2018;14(5):285–300. doi:10.1038/nrendo.2018.22 - DOI - PubMed
    1. Dahlgren J, Noordam C. Growth, endocrine features, and growth hormone treatment in Noonan syndrome. J Clin Med. 2022;11(7):2034. doi:10.3390/jcm11072034 - DOI - PMC - PubMed
    1. European Medicines Agency [Internet]. Omnitrope® European public assessment report (EPAR), updated 2018; 2008. Available from: https://www.ema.europa.eu/en/documents/overview/omnitrope-epar-summary-p.... Accessed June 30, 2023.

MeSH terms