Comparison of response to 2-years' growth hormone treatment in children with isolated growth hormone deficiency, born small for gestational age, idiopathic short stature, or multiple pituitary hormone deficiency: combined results from two large observational studies.

Authors

Peter A Lee, Penn State College of Medicine, The Milton S. Hershey Medical CenterFollow
Lars Sävendahl, Division of Pediatric Endocrinology, Department of Women’s and Children´s Health, Karolinska InstitutetFollow
Isabelle Oliver, Division of Pediatric Endocrinology, Hôpital des Enfants
Maithé Tauber, Division of Pediatric Endocrinology, Hôpital des Enfants
Oliver Blankenstein, Department of Pediatric Endocrinology, Charité-Universitätsmedizin Berlin
Judith Ross, Department of Pediatrics, Thomas Jefferson UniversityFollow
Marta Snajderova, Department of Pediatrics, 2nd Medical School - Charles University and University Hospital Motol
Viatcheslav Rakov, Novo Nordisk Health Care AG
Birgitte Tønnes Pedersen, Novo Nordisk A/SFollow
Henrik Thybo Christesen, H.C. Andersen Children’s Hospital, Odense University HospitalFollow

Document Type

Article

Publication Date

7-12-2012

Comments

This article has been peer reviewed. It was published in: International journal of pediatric endocrinology.

2012; 2012(1): 22.

The published version is available at http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3441784/. DOI: 10.1186/1687-9856-2012-22.

Copyright © 2012 Lee et al.; licensee BioMed Central Ltd.

Abstract

BACKGROUND: Few studies have compared the response to growth hormone (GH) treatment between indications such as isolated growth hormone deficiency (IGHD), born small for gestational age (SGA), idiopathic short stature (ISS), and multiple pituitary hormone deficiency (MPHD). The aim of this analysis of data, collected from two large ongoing observational outcome studies, was to evaluate growth and insulin-like growth factor-I (IGF-I) response data for children of short stature with IGHD, MPHD, SGA, or ISS following two years of treatment with the recombinant GH product Norditropin® (Novo Nordisk A/S, Bagsværd, Denmark).

METHODS: Analysis of auxologic data from two ongoing prospective observational studies, NordiNet® International Outcomes Study (NordiNet® IOS) and NovoNet®/American Norditropin® Studies: Web-enabled Research (ANSWER) Program®.

RESULTS: 4,582 children agedIGHD, n = 3,298; SGA, n = 678; ISS, n = 334; and MPHD, n = 272. After two years' GH treatment, change in height standard deviation score (SDS) was +1.03 in SGA and +0.84 in ISS vs. +0.97 in IGHD (p = 0.047; p < 0.001 vs. IGHD, respectively). Height gain was comparable between IGHD and MPHD. In pre-pubertal children vs. total population, height SDS change after two years was: IGHD, +1.24 vs. +0.97; SGA, +1.17 vs. +1.03; ISS, +1.04 vs. +0.84; and MPHD, +1.16 vs. +0.99 (all p < 0.001).

CONCLUSIONS: After two years' GH treatment, change in height SDS was greater in SGA and less in ISS, compared with IGHD; the discrepancy in responses may be due to the disease nature or confounders (i.e. age). Height SDS increase was greatest in pre-pubertal children, supporting early treatment initiation to optimize growth outcomes.

Recommended Citation

Lee, Peter A; Sävendahl, Lars; Oliver, Isabelle; Tauber, Maithé; Blankenstein, Oliver; Ross, Judith; Snajderova, Marta; Rakov, Viatcheslav; Pedersen, Birgitte Tønnes; and Christesen, Henrik Thybo, "Comparison of response to 2-years' growth hormone treatment in children with isolated growth hormone deficiency, born small for gestational age, idiopathic short stature, or multiple pituitary hormone deficiency: combined results from two large observational studies." (2012). Department of Pediatrics Faculty Papers. Paper 53.
https://jdc.jefferson.edu/pedsfp/53

PubMed ID

22788856