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Safety and effectiveness of a somatropin biosimilar in children requiring growth hormone treatment: second analysis of the PATRO Children study Italian cohort

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Abstract

Purpose

To investigate the long-term safety (primary endpoint) and effectiveness (secondary endpoint) of the somatropin biosimilar Omnitrope®.

Methods

PATRO Children is an ongoing, multicenter, observational, post-marketing surveillance study. Children who received Omnitrope® for any indication were included. Adverse events (AEs) were evaluated in all study participants. Auxological data, including height standard deviation scores (HSDS) and height velocity standard deviation scores (HVSDS), were used to assess effectiveness. In this snapshot analysis, data from the Italian subpopulation up to August 2017 were reported.

Results

A total of 291 patients (mean age 10.0 years, 56.0% male) were enrolled at 19 sites in Italy. The mean duration of Omnitrope® treatment was 33.1 ± 21.7 months. There were 48 AEs with a suspected relationship to the study drug (as reported by the investigator) that occurred in 35 (12.0%) patients, most commonly headache, pyrexia, arthralgia, insulin-like growth factor above normal range, abdominal pain, pain in extremity and acute gastroenteritis. There were no confirmed cases of type 1 or type 2 diabetes; however, two patients (0.7%) had impaired glucose tolerance that was considered Omnitrope® related. The mean HSDS increased from − 2.41 ± 0.73 at baseline (n = 238) to − 0.91 ± 0.68 at 6.5 years (n = 10). The mean HVSDS increased from − 1.77 ± 1.38 at baseline (n = 136) to 0.96 ± 1.13 at 6.5 years (n = 10).

Conclusions

In this sub-analysis of PATRO Children, Omnitrope® appeared to have acceptable safety and effectiveness in the treatment of in Italian children, which was consistent with the earlier findings from controlled clinical trials.

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Data availability

The datasets generated and/or analyzed during the current study are available from the corresponding author on reasonable request.

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Acknowledgements

Medical writing assistance in drafting the outline and first draft of this manuscript was provided by Matt Shirley and Mimi Chan, PhD, of Springer Healthcare Communications, respectively. This medical writing assistance was funded by Sandoz, Italy.

Funding

The study was funded by Sandoz, Italy.

Author information

Authors and Affiliations

  1. Pediatric Unit, Department of Medical and Surgical Sciences for Mother, Children and Adults, University of Modena and Reggio Emilia, Via del Pozzo, 41124, Modena, Italy

    L. Iughetti

  2. UO di Pediatria, Ospedale Donna e Bambino, Dipartimento di Scienze Chirurgiche, Odontostomatologiche e Materno Infantili, Università di Verona, Verona, Italy

    F. Antoniazzi

  3. Unità Operativa di Endocrinologia, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico di Milano, Dipartimento di Scienze Cliniche e di Comunità, Università di Milano, Milan, Italy

    C. Giavoli

  4. Dipartimento di Scienze della Salute, Università del Piemonte Orientale, Novara, Italy

    G. Bona

  5. Department of Human Pathology in Adulthood and Childhood, University of Messina, Messina, Italy

    T. Aversa

  6. UO di Endocrinologia Pediatrica e Adolescentologia, Dip. Salute della Donna e del Bambino, Az. Ospedaliera-Università di Padova, Padua, Italy

    N. A. Greggio

  7. Unita Operativa di Endocrinologia Pediatrica e Adolescentologia Clinica Pediatrica-Università Degli Studi di Padova Azienda Ospedaliera di Padova, Padua, Italy

    L. Guazzarotti

  8. Clinica Pediatrica, Dipartimento Materno Infantile, Azienda Ospedaliero-Universitaria di Parma, Parma, Italy

    R. Minelli

  9. Dipartimento Della Donna, del Bambino e di Chirurgia Generale e Specialistica, Università Degli Studi Luigi Vanvitelli, Naples, Italy

    L. Perrone

  10. Division of Endocrine and Metabolic Diseases, IRCCS Istituto Auxologico Italiano, Milan, Italy

    L. Persani

  11. Department of Clinical Sciences and Community Health, University of Milan, Milan, Italy

    L. Persani

  12. Clinica Pediatrica Dipartimento Materno-Infantile IRCCS Ospedale San Raffaele Università Vita-Salute San Raffaele, Milan, Italy

    G. Pozzobon

  13. Dipartimento Per Il Ritardo Mentale, Unità Operativa di Pediatria e Genetica Medica, Oasi Research Institute-I.R.C.C.S., Troina, Enna, Italy

    L. Ragusa

  14. Clinica Pediatrica I, Auxoendocrinologia e Ginecologia Pediatrica, AOU Meyer, Florence, Italy

    S. Stagi

  15. Clinica Pediatrica Ospedale “Burlo Garofalo” di Trieste, Trieste, Italy

    G. Tornese

  16. Dipartimento di Scienze Biomediche Ed Oncologia Umana, AO Policlinico di Bari Ospedale Giovanni XXIII, Bari, Italy

    C. Zecchino

  17. Sandoz S.P.A, Origgio, Italy

    P. Gallinari & P. Fedeli

  18. Sandoz Biopharmaceutical C/O HEXAL AG, Holzkirchen, Germany

    H. Zouater

  19. Unit of Pediatric Endocrinology, S.Orsola-Malpighi Hospital, Bologna, Italy

    S. Zucchini

Authors
  1. L. Iughetti
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  2. F. Antoniazzi
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  3. C. Giavoli
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  4. G. Bona
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  5. T. Aversa
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  6. N. A. Greggio
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  7. L. Guazzarotti
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  8. R. Minelli
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  9. L. Perrone
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  10. L. Persani
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  12. L. Ragusa
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Contributions

LP, LP, and NAG enrolled patients, and read and approved the manuscript drafts. SZ and LI contributed to study design, enrolled patients, and critically revised the manuscript.FA, GB, TA, LG, RM, GP, LR, SS, GT, PG, HZ, PF, and CZ contributed to the drafting of the manuscript, critically revised the various drafts of the manuscript, read and approved the final version before submission. CG enrolled patients, critically revised the various drafts of the manuscript, and read and approved the final version before submission.

Corresponding author

Correspondence to L. Iughetti.

Ethics declarations

Conflict of interest

LI has participated in Advisory Boards for Eli Lilly, Italy and Novo Nordisk, Italy; FA has received honoraria from Eli Lilly, Novo Nordisk and Ipsen and research funding from Merck Serono; CG, TA, NAG, LG, RM, L Perrone, LR, SS, and GT have no conflict of interest to declare; L Persani has been an invited speaker for Sandoz and Merck-Serono, and has received unconditional research funds from Novartis, IBSA and Merck-Serono; GB has received honoraria from Ferring, Novo, Serono, Lilly, Sandoz; CZ has received honoraria from Sandoz, Novo, Medtronic; HZ is an employee of Sandoz Biopharmaceutical c/o HEXAL AG, Germany; PG and PF are employees of Sandoz S.p.A, Milan, Italy; SZ has been a member of expert committees for Eli Lilly Diabetes, Italy, and Roche Diagnostics.

Ethical approval

All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee (Comitato Etico Indipendente dell’Azienda Ospedaliero-Universitaria di Bologna, Policlinico S. Orsola-Malpighi, n°120/2007/O/Oss) and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards.

Informed consent

Informed consent was obtained from the parents or legal guardians of all individual participants included in the study.

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Iughetti, L., Antoniazzi, F., Giavoli, C. et al. Safety and effectiveness of a somatropin biosimilar in children requiring growth hormone treatment: second analysis of the PATRO Children study Italian cohort. J Endocrinol Invest 44, 493–503 (2021). https://doi.org/10.1007/s40618-020-01331-4

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  • DOI: https://doi.org/10.1007/s40618-020-01331-4

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