Outcomes in children treated with growth hormone for Prader-Willi syndrome: data from the ANSWER Program® and NordiNet® International Outcome Study
| العنوان: | Outcomes in children treated with growth hormone for Prader-Willi syndrome: data from the ANSWER Program® and NordiNet® International Outcome Study |
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| المؤلفون: | Alberto Pietropoli, Nicky Kelepouris, Vlady Ostrow, Moris Angulo, Maithé Tauber, M. Jennifer Abuzzahab |
| المساهمون: | New York University Langone Medical Center (NYU Langone Medical Center), NYU System (NYU), Children’s Minnesota Hospital [Minneapolis], University hospital of Zurich [Zurich], Novo nordisk pharma, Novo Nordisk Inc, NOVO NORDISK PARK, Centre de Physiopathologie Toulouse Purpan (CPTP), Université Toulouse III - Paul Sabatier (UT3), Université Fédérale Toulouse Midi-Pyrénées-Université Fédérale Toulouse Midi-Pyrénées-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Novo Nordisk |
| المصدر: | International Journal of Pediatric Endocrinology, Vol 2020, Iss 1, Pp 1-8 (2020) International Journal of Pediatric Endocrinology International Journal of Pediatric Endocrinology, BioMed Central, 2020, 2020 (1), pp.20. ⟨10.1186/s13633-020-00090-6⟩ |
| بيانات النشر: | BMC, 2020. |
| سنة النشر: | 2020 |
| مصطلحات موضوعية: | 0301 basic medicine, Pediatrics, medicine.medical_specialty, Body height, 030209 endocrinology & metabolism, Growth disorders, Growth hormone, lcsh:Diseases of the endocrine glands. Clinical endocrinology, 03 medical and health sciences, 0302 clinical medicine, [SDV.MHEP.MI]Life Sciences [q-bio]/Human health and pathology/Infectious diseases, medicine, In patient, Registries, Body mass index, 2. Zero hunger, lcsh:RC648-665, Human growth hormone, business.industry, Research, lcsh:RJ1-570, nutritional and metabolic diseases, lcsh:Pediatrics, Treatment period, 3. Good health, Somatropin, 030104 developmental biology, Gh treatment, Observational study, business |
| الوصف: | BackgroundGrowth hormone (GH) deficiency is common in patients with Prader-Willi syndrome (PWS) and leads to short adult stature. The current study assessed clinical outcomes based on real-world observational data in pediatric patients with PWS who were treated with GH.MethodsData from patients previously naïve to treatment with GH who began therapy with somatropin were collected from 2006 to 2016 in the observational American Norditropin® Studies: Web-Enabled Research (ANSWER) Program® and NordiNet® International Outcome Study. Variables affecting change from baseline in height standard deviation scores (HSDS;n = 129) and body mass index standard deviation scores (BMI SDS;n = 98) were determined.ResultsPatients included in both HSDS and BMI SDS analyses were treated with a mean GH dose of 0.03 mg/kg/d (SD, 0.01 mg/kg/d). Results from the HSDS analysis revealed that baseline age and years on treatment had a significant impact on the change in HSDS. In the BMI SDS analysis, longer GH treatment time led to a greater change in BMI SDS from baseline, and patients with a higher BMI at the start of treatment had a greater decrease in BMI over time.ConclusionsGH is effective in the management of children with PWS. Earlier treatment resulted in a greater gain in height, and a longer treatment period resulted in better outcomes for both height and BMI.Trial registrationThis study was registered with ClinicalTrials.gov (NCT01009905) on November 9, 2009. |
| اللغة: | English |
| تدمد: | 1687-9856 |
| DOI: | 10.1186/s13633-020-00090-6 |
| DOI: | 10.1186/s13633-020-00090-6⟩ |
| URL الوصول: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::25cbd8cd9ae022df8b85a4b6ab8ae4f7 http://link.springer.com/article/10.1186/s13633-020-00090-6 |
| Rights: | OPEN |
| رقم الانضمام: | edsair.doi.dedup.....25cbd8cd9ae022df8b85a4b6ab8ae4f7 |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 16879856 |
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| DOI: | 10.1186/s13633-020-00090-6 |