Tilman R Rohrer1, Sabine Ceplis-Kastner2, Norbert Jorch3, Hermann L Müller4, Roland Pfäffle5, Thomas Reinehr6, Annette Richter-Unruh7, Claudia Weißenbacher8, Paul-Martin Holterhus9, Dr Sabine Clips-Kastner Ferring Arzneimittel GmbH. 1. Department of Paediatrics, Saarland University Medical Centre, Homburg/Saar, Germany. 2. Medical Department, Ferring Arzneimittel GmbH, Kiel, Germany. 3. Protestant Hospital of the Bethel Foundation, Children's Hospital, Bielefeld, Germany. 4. Department of Paediatrics and Paediatric Haematology/Oncology, University Children's Hospital, Klinikum Oldenburg AöR, Oldenburg, Germany. 5. Department of Paediatric Endocrinology, Children's Hospital, Leipzig, Germany. 6. Department of Paediatric Endocrinology, Diabetes, and Nutrition Medicine, Vestische Kinder- und Jugendklinik, University of Witten/Herdecke, Datteln, Germany. 7. Department of Paediatric Endocrinology and Diabetology, University Children's Hospital, Ruhr University Bochum, Bochum, Germany. 8. Department of Endocrinology, Dr. von Haunersches Children's Hospital, LMU Munich, Munich, Germany. 9. Division of Paediatric Endocrinology and Diabetes, Christian-Albrechts University of Kiel (CAU) and University Hospital Schleswig-Holstein (UKSH), Campus Kiel, Kiel, Germany, PaulMartin.Holterhus@uksh.de.
Abstract
BACKGROUND: Treatment with growth hormone (GH) is standard clinical practice in children with GH deficiency (GHD) or Turner syndrome (TS). Hitherto, no long-term data on auxological outcome and safety of Zomacton® have been published. Data comparing needle-free administration (NF) and needle injection (NI) of GH are very sparse. AIMS: To analyse longitudinal auxological outcome and safety data of GH treatment-naïve patients diagnosed with GHD or TS and to compare NF and NI in a real-life setting. METHODS: Pooled auxological data and safety information from three consecutive prospective observational Zomacton® studies covering 22 years of treatment were analysed and NF was compared to NI. RESULTS: The safety cohort comprised 1,595 patients who received at least one GH dose. The auxological outcome cohort comprised 856 treatment-naïve patients with follow-up data ≥12 months. Height-SDS and height velocity improved significantly during the first 3 years of treatment. Documented choice of device was available for 658 patients (NF 69.1%, NI 30.9%). NF administration was non-inferior to NI. No previously unknown safety signals occurred. CONCLUSION: Real-life data show that treatment with Zomacton® improves auxological outcome parameters without new safety concerns. NF administration of GH represents a useful alternative to NI in children with growth disorders.
BACKGROUND: Treatment with growth hormone (GH) is standard clinical practice in children with GH deficiency (GHD) or Turner syndrome (TS). Hitherto, no long-term data on auxological outcome and safety of Zomacton® have been published. Data comparing needle-free administration (NF) and needle injection (NI) of GH are very sparse. AIMS: To analyse longitudinal auxological outcome and safety data of GH treatment-naïve patients diagnosed with GHD or TS and to compare NF and NI in a real-life setting. METHODS: Pooled auxological data and safety information from three consecutive prospective observational Zomacton® studies covering 22 years of treatment were analysed and NF was compared to NI. RESULTS: The safety cohort comprised 1,595 patients who received at least one GH dose. The auxological outcome cohort comprised 856 treatment-naïve patients with follow-up data ≥12 months. Height-SDS and height velocity improved significantly during the first 3 years of treatment. Documented choice of device was available for 658 patients (NF 69.1%, NI 30.9%). NF administration was non-inferior to NI. No previously unknown safety signals occurred. CONCLUSION: Real-life data show that treatment with Zomacton® improves auxological outcome parameters without new safety concerns. NF administration of GH represents a useful alternative to NI in children with growth disorders.
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