Multicenter Study

. 2025 Mar;87(3):933-942.

doi: 10.1007/s12020-024-04090-x. Epub 2024 Nov 29.

Long-term safety and effectiveness of a somatropin biosimilar (Omnitrope^®) in children requiring growth hormone therapy: analysis of final data of Italian patients enrolled in the PATRO children study

Lorenzo Iughetti¹, Franco Antoniazzi², Claudia Giavoli³, Simonetta Bellone⁴, Tommaso Aversa⁵, Laura Guazzarotti⁶, Maria Elisabeth Street⁷, Emanuele Miraglia Del Giudice⁸, Luca Persani^{9

10}, Gabriella Pozzobon¹¹, Letizia Ragusa¹², Stefano Stagi^{13

14}, Gianluca Tornese^{15

16}, Clara Zecchino¹⁷, Chiara Mameli¹⁸, Emiliano Zecchi¹⁹, Paolo Fedeli¹⁹, Markus Zabransky²⁰, Laura Lucaccioni²¹, Stefano Zucchini²²

Affiliations

¹ Pediatric Unit, Department of Medical and Surgical Sciences for Mother, Children and Adults, University of Modena and Reggio Emilia, Modena, Italy. lorenzo.iughetti@unimore.it.
² Pediatric Clinic C, Department of Surgical Sciences, Dentistry, Gynecology and Pediatrics, University of Verona, Verona, Italy.
³ Endocrinology Unit, Fondazione IRCCS Cà Granda, Ospedale Maggiore Policlinico, Milan, Italy.
⁴ Division of Pediatrics, Department of Health Sciences, University of Piemonte Orientale, Novara, Italy.
⁵ Pediatric Unit, Department of Human Pathology of Adulthood and Childhood "G. Barresi", University of Messina, Messina, Italy.
⁶ Pediatric Endocrinology Unit, University of Padova, Padova, Italy.
⁷ Department of Medicine and Surgery, Unit of Paediatrics, University Hospital of Parma, Parma, Italy.
⁸ Department of General and Specialized Surgery for Women and Children, Università degli Studi Luigi Vanvitelli, Napoli, Italy.
⁹ Division of Endocrine and Metabolic Diseases, IRCCS Istituto Auxologico Italiano, Milan, Italy.
¹⁰ Department of Medical Biotechnology and Translational Medicine, University of Milan, Milan, Italy.
¹¹ Paediatric Department San Raffaele Hospital, Vita-Salute University San Raffaele, Milan, Italy.
¹² Department of Intellectual Disabilities, Pediatric and Medical Genetics Unit, Oasi Research Institute-I.R.C.C.S., Troina, Enna, Italy.
¹³ Health Sciences Department, University of Florence, Florence, Italy.
¹⁴ Auxoendocrinologia, Ospedale Pediatrico Meyer IRCCS, Florence, Italy.
¹⁵ Institute for Maternal and Child Health IRCCS Burlo Garofolo, Trieste, Italy.
¹⁶ Department of Medicine, Surgery and Health Sciences, University of Trieste, Trieste, Italy.
¹⁷ Department of Biomedical Sciences and Human Oncology, AO Policlinico of Bari, Giovanni XXIII Hospital, Bari, Italy.
¹⁸ Department of Pediatrics, V. Buzzi Children Hospital, University of Milan, Milan, Italy.
¹⁹ Sandoz S.p.A, Milan, Italy.
²⁰ Sandoz Biopharmaceutical c/o HEXAL AG, Holzkirchen, Germany.
²¹ Pediatric Unit, Department of Medical and Surgical Sciences for Mother, Children and Adults, University of Modena and Reggio Emilia, Modena, Italy.
²² Pediatric Unit, IRCCS AOU of Bologna, Bologna, Italy.

PMID: 39612101
DOI: 10.1007/s12020-024-04090-x

Multicenter Study

Long-term safety and effectiveness of a somatropin biosimilar (Omnitrope^®) in children requiring growth hormone therapy: analysis of final data of Italian patients enrolled in the PATRO children study

Lorenzo Iughetti et al. Endocrine. 2025 Mar.

. 2025 Mar;87(3):933-942.

doi: 10.1007/s12020-024-04090-x. Epub 2024 Nov 29.

Authors

Affiliations

¹ Pediatric Unit, Department of Medical and Surgical Sciences for Mother, Children and Adults, University of Modena and Reggio Emilia, Modena, Italy. lorenzo.iughetti@unimore.it.
² Pediatric Clinic C, Department of Surgical Sciences, Dentistry, Gynecology and Pediatrics, University of Verona, Verona, Italy.
³ Endocrinology Unit, Fondazione IRCCS Cà Granda, Ospedale Maggiore Policlinico, Milan, Italy.
⁴ Division of Pediatrics, Department of Health Sciences, University of Piemonte Orientale, Novara, Italy.
⁵ Pediatric Unit, Department of Human Pathology of Adulthood and Childhood "G. Barresi", University of Messina, Messina, Italy.
⁶ Pediatric Endocrinology Unit, University of Padova, Padova, Italy.
⁷ Department of Medicine and Surgery, Unit of Paediatrics, University Hospital of Parma, Parma, Italy.
⁸ Department of General and Specialized Surgery for Women and Children, Università degli Studi Luigi Vanvitelli, Napoli, Italy.
⁹ Division of Endocrine and Metabolic Diseases, IRCCS Istituto Auxologico Italiano, Milan, Italy.
¹⁰ Department of Medical Biotechnology and Translational Medicine, University of Milan, Milan, Italy.
¹¹ Paediatric Department San Raffaele Hospital, Vita-Salute University San Raffaele, Milan, Italy.
¹² Department of Intellectual Disabilities, Pediatric and Medical Genetics Unit, Oasi Research Institute-I.R.C.C.S., Troina, Enna, Italy.
¹³ Health Sciences Department, University of Florence, Florence, Italy.
¹⁴ Auxoendocrinologia, Ospedale Pediatrico Meyer IRCCS, Florence, Italy.
¹⁵ Institute for Maternal and Child Health IRCCS Burlo Garofolo, Trieste, Italy.
¹⁶ Department of Medicine, Surgery and Health Sciences, University of Trieste, Trieste, Italy.
¹⁷ Department of Biomedical Sciences and Human Oncology, AO Policlinico of Bari, Giovanni XXIII Hospital, Bari, Italy.
¹⁸ Department of Pediatrics, V. Buzzi Children Hospital, University of Milan, Milan, Italy.
¹⁹ Sandoz S.p.A, Milan, Italy.
²⁰ Sandoz Biopharmaceutical c/o HEXAL AG, Holzkirchen, Germany.
²¹ Pediatric Unit, Department of Medical and Surgical Sciences for Mother, Children and Adults, University of Modena and Reggio Emilia, Modena, Italy.
²² Pediatric Unit, IRCCS AOU of Bologna, Bologna, Italy.

PMID: 39612101
DOI: 10.1007/s12020-024-04090-x

Erratum in

Correction to: Long-term safety and effectiveness of a somatropin biosimilar (Omnitrope®) in children requiring growth hormone therapy: analysis of final data of Italian patients enrolled in the PATRO children study.
Iughetti L, Antoniazzi F, Giavoli C, Bellone S, Aversa T, Guazzarotti L, Street ME, Miraglia Del Giudice E, Persani L, Pozzobon G, Ragusa L, Stagi S, Tornese G, Zecchino C, Mameli C, Zecchi E, Fedeli P, Zabransky M, Lucaccioni L, Zucchini S. Iughetti L, et al. Endocrine. 2025 May;88(2):668-669. doi: 10.1007/s12020-025-04162-6. Endocrine. 2025. PMID: 39883391 No abstract available.

Abstract

Purpose: Omnitrope^® (a somatropin biosimilar), used to treat growth disturbances, is considered to have a good safety profile in children. Here, we present the analysis of final data of the Italian cohort of the PAtients TReated with Omnitrope^® (PATRO) Children study.

Methods: This multicenter, open-label, longitudinal, post-marketing surveillance study enrolled eligible children during 2010-2018. The primary objective was to assess the long-term safety of Omnitrope^® by recording all adverse events (AEs), serious AEs, and adverse drug reactions (ADRs). A secondary objective was to evaluate the long-term effectiveness of Omnitrope^® using height measurements.

Results: A total of 375 patients were included in the Italian cohort of the PATRO Children study. After a mean ± standard deviation (SD) follow-up duration of 40.9 ± 24.6 months, 607 AEs were reported in 58.4% of patients, mostly of mild (52.5%) or moderate (15.7%) severity. The most common AEs were headache (11.7%), elevated insulin-like growth factor (IGF)-1 (4.8%), abdominal pain (4.3%), and pyrexia (3.7%). Sixty-seven ADRs occurred in 52 patients (13.9%); the most common ADRs were elevated IGF-1 (3.5%) and insulin resistance (2.9%). Mean ± SD height standard deviation scores in treatment-naïve patients increased from -2.5 ± 0.7 at baseline (n = 318) to -1.3 ± 0.7 at 5 years (n = 56) and to -0.8 ± 0.7 at 7.5 years (n = 13).

Conclusions: This final analysis extends the interim analysis findings from the PATRO Children study and confirms the long-term safety and effectiveness of Omnitrope^® in Italian pediatric patients with growth disturbances.

Keywords: Adolescents; Children; Infants; Omnitrope®; Pediatric; Recombinant human growth hormone.

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Conflict of interest statement

Compliance with ethical standards. Conflict of interest: Lorenzo Iughetti has received fees for participation on advisory boards from Sandoz. Franco Antoniazzi has received research funding from Merck Serono and Sandoz, and fees for participation on advisory boards from BioMarin. Tommaso Aversa has consulted for Pfizer and Sandoz. Luca Persani has received fees for participation on advisory boards from Sandoz. Gabriella Pozzobon has received consulting fees and for participation on advisory boards from Sandoz, Merck Serono and Novo Nordisk. Emiliano Zecchi, Paolo Fedeli and Markus Zabransky are part of Sandoz Company Organization. Claudia Giavoli, Simonetta Bellone, Laura Guazzarotti, Maria Elisabeth Street, Emanuele Miraglia del Giudice, Stefano Stagi, Gianluca Tornese, Chiara Mameli, Laura Lucaccioni, Letizia Ragusa, Clara Zecchino, and Stefano Zucchini have no relevant financial or non-financial interests to disclose. Ethics approval: The study was reviewed and approved by each study site’s Independent Ethics Committee/Institutional Review Board. Research involving human participants: All procedures performed in this study involving human participants were in accordance with the ethical standards of the institutional and/or national research committees, with the 1964 Helsinki Declaration and its later amendments, and with the ethical principles outlined in the Oviedo Human Rights Convention. Consent to participate: All patients (or their parents/legal guardians) provided written informed consent before study inclusion.

References

1. European Medicines Agency. Omnitrope 1.3 mg/mL powder and solvent for solution for injection [summary of product characteristics]. 2015. Available from: https://www.ema.europa.eu/en/documents/product-information/omnitrope-epa... . Accessed: 5 Dec 2022.
1. US Food and Drug Administration. Omnitrope^® (somatropin) injection, for subcutaneous use [prescribing information]. 2016. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/021426s022lbl.pdf . Accessed: 5 Dec 2022.
1. D.B. Allen, P. Backeljauw, M. Bidlingmaier, B.M. Biller, M. Boguszewski, P. Burman et al. GH safety workshop position paper: a critical appraisal of recombinant human GH therapy in children and adults. Eur. J. Endocrinol. 174(2), P1–P9 (2016). https://doi.org/10.1530/EJE-15-0873 - DOI - PubMed
1. A.J. Swerdlow, R. Cooke, D. Beckers, B. Borgstrom, G. Butler, J.C. Carel et al. Cancer risks in patients treated with growth hormone in childhood: the SAGhE European cohort study. J. Clin. Endocrinol. Metab. 102(5), 1661–1672 (2017). https://doi.org/10.1210/jc.2016-2046 - DOI - PubMed
1. S. Cianfarani, Safety of pediatric rhGH therapy: an overview and the need for long-term surveillance. Front Endocrinol 12, 811846 (2021). https://doi.org/10.3389/fendo.2021.811846 - DOI

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Long-term safety and effectiveness of a somatropin biosimilar (Omnitrope^®) in children requiring growth hormone therapy: analysis of final data of Italian patients enrolled in the PATRO children study

Affiliations

Long-term safety and effectiveness of a somatropin biosimilar (Omnitrope^®) in children requiring growth hormone therapy: analysis of final data of Italian patients enrolled in the PATRO children study

Authors

Affiliations

Erratum in

Abstract

Conflict of interest statement

References

Publication types

MeSH terms

Substances

LinkOut - more resources

Full Text Sources

Medical

Miscellaneous