Abstract
Context: Understanding real-world prescribing of GH may help improve treatment of eligible patients. Objective: Overall: to assess real-world effectiveness and safety of GH (Norditropin). This analysis: to compare clinical characteristics of GH-treated children in the United States and Europe. Design: The American Norditropin Studies: Web-Enabled Research Program (ANSWER; 2002 to 2016, United States) and the NordiNet International Outcome Study (NordiNet IOS; 2006 to 2016, Europe) were multicenter longitudinal observational cohort studies. Setting: Data were recorded in 207 (United States) and 469 (Europe) clinics. Participants: Patients with GH deficiency, Turner syndrome, Noonan syndrome, idiopathic short stature, Prader-Willi syndrome, or born small for gestational age, who commenced GH treatment aged <1 year. Intervention: GH was prescribed by treating physicians according to local practice. Main Outcomes Measures: Baseline data and drug doses were recorded. Data on effectiveness and safety were collected. Results: ANSWER had 19,847 patients in the full analysis set (FAS; patients with birthdate information and one or more GH prescription) and 12,660 in the effectiveness analysis set (EAS; GH-naive patients with valid baseline information). NordiNet IOS had 17,711 (FAS) and 11,967 (EAS). Boys accounted for 69% (ANSWER) and 57% (NordiNet IOS). Treatment start occurred later than optimal to improve growth. The proportion of boys treated was generally larger, children were older at treatment start, and GH doses were higher in the United States vs Europe. No new safety signals of concern were noted. Conclusions: In most indications, more boys than girls were treated, and treatment started late. Earlier diagnosis of GH-related disorders is needed. The data support a favorable benefit-risk profile of GH therapy in children.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 4730-4742 |
| Number of pages | 13 |
| Journal | Journal of Clinical Endocrinology and Metabolism |
| Volume | 104 |
| Issue number | 10 |
| DOIs | |
| State | Published - Oct 1 2019 |
Bibliographical note
Funding Information:The authors thank all the investigators, as well as patients and their families, who participated in the two studies. We would like to warmly thank Birgitte Tønnes Pedersen for valuable input to the manuscript. Data analysis was performed by Birgitte Tønnes Pedersen, employee of Novo Nordisk A/S at the time, and Jean-Marc Ferran (Qualiance ApS) under contract to Novo Nordisk A/S. We also thank Jean-Marc Ferran and Navid Nedjatian (Novo Nordisk) for review and input to the manuscript. Medical writing and submission support were provided by Grace Townshend, Penny Butcher, Germanicus Hansa-Wilkinson, and Helen Marshall of Watermeadow Medical, funded by Novo Nordisk Health Care AG. These results have been partially presented at the 20th European Congress of Endocrinology, Barcelona, Spain, 19–22 May 2018; 57th Annual Meeting of the European Society for Paediatric Endocrinology, Athens, Greece, 27–29 September 2018; XXVII Latin American Congress of Pediatric Endocrinology, Cusco, Peru, 24–27 October 2018; and 10th Asia Pacific Paediatric Endocrine Society (APPES) Biennial Scientific Meeting, Chiang Mai, Thailand, 7–10 November 2018.
Funding Information:
Disclosure Summary: L.S. is President of the NordiNet IOS Committee and has received consultation fees from Ascendis, Novo Nordisk, Merck, Pfizer, and Sandoz. M.P. consulted for, and received honoraria, travel grants, and unrestricted research grants from Novo Nordisk; was on the Advisory Board for the Ipsen’s Increlex registry, on an Advisory Board for Pfizer, and advised for Novo Nordisk GNAP; and has received research grants from Novo Nordisk, Merck, Ipsen, Pfizer, and Sandoz. P.B. has received research funding from Novo Nordisk and has been a consultant for Novo Nordisk, including advisory board participation. J.B. has received payment for roles on the NordiNet IOS Steering Committee and Publication Steering Committee; has received honoraria from Novo Nordisk and Sandoz for presentations at academic meetings; and has received financial support in the form of registration fees from Novo Nordisk to attend academic meetings. B.S.M. is a consultant for AbbVie, Ascendis, Ferring, Novo Nordisk, Pfizer, Sandoz, Soleno, and Tolmar and has received research support from Alexion, Ascendis, BioMarin, Endo Pharmaceuticals, Genentech, Genzyme, Novo Nordisk, Opko, Sandoz, Sangamo, Shire, Tolmar, and Versartis. T.R.R. has acted as a consultant for Novo Nordisk and received speaker honoraria from Novo Nordisk. A.P. and V.O. are employees of Novo Nordisk. J.R. is a consultant for Novo Nordisk and Opko and receives research funding from Novo Nordisk.
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