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01.12.2016 | Research | Ausgabe 1/2016 Open Access

International Journal of Pediatric Endocrinology 1/2016

Efficacy of growth hormone replacement on anthropometric outcomes, obesity, and lipids in children with optic nerve hypoplasia and growth hormone deficiency

International Journal of Pediatric Endocrinology > Ausgabe 1/2016
Carly Stewart, Pamela Garcia-Filion, Cassandra Fink, Anna Ryabets-Lienhard, Mitchell E. Geffner, Mark Borchert
Wichtige Hinweise

Competing interests

MG is a clinical trial consultant with Daiichi-Sankyo; is a member of the Data Safety Monitoring Board for Tolmar; has research contracts with Novo Nordisk and Versartis; has research grants from Eli Lilly and Company, Genentech, and Novo Nordisk; receives royalties from McGraw-Hill and UpToDate; and is on advisory boards for Endo Pharmaceuticals, Ipsen, Pfizer, and Sandoz. MB held a research grant from Genentech. Authors CS, PGF, CF, and AR declare that they have no competing interests.

Authors’ contributions

CS served as study coordinator, collected patient data, performed literature review and descriptive statistics, and drafted the manuscript. PGF was involved in initial study conception and design, performed analysis and interpretation of data, and helped design, draft, and provide critical revisions to the manuscript. CF served as an earlier study coordinator, was involved in study design, data collection, and critical revision of the manuscript. AR served as study endocrinologist, helped interpret data, and provided critical revision to the manuscript. As co-investigator, MG provided guidance on clinical relevance of findings, and contributed to the design, intellectual content, and critical revision of the manuscript. As principal investigator, MB was involved in study conception, design and execution, aided in the interpretation of findings, and contributed to the design, intellectual content, and critical revision of the manuscript. All authors read and approved the final manuscript.



Hypopituitarism and obesity are causes of major lifelong morbidity in patients with optic nerve hypoplasia (ONH). Growth hormone deficiency (GHD) affects the majority of children with ONH, though the degree of deficiency and variability of early growth patterns range from early severe retardation to normal initial growth. The utility of early GH replacement for improving anthropometric, body composition, and lipid outcomes in patients with ONH and GHD, especially those with normal initial height velocity, is unknown. This study examines the effects of GH replacement in a cohort of children with ONH and GHD.


Controlled clinical trial from 2005–2014. The study included 17 children with ONH and untreated GHD. Those meeting criteria for growth deceleration were assigned to treatment with recombinant human growth hormone (n = 5) while those with normal height velocity were randomized either to treatment (n = 5) or to observation (no intervention, n = 7). Study duration was 3 years. Primary outcome measures included stature, weight, weight-for-stature, and BMI standard deviation score (SDS) at study completion.


Subjects on GH, irrespective of entry growth trajectory, grew more on average in stature than controls by a difference of 0.98 SDS by study end; this effect persisted after adjusting for baseline overweight status. Treatment had an effect on weight SDS only after adjusting for initial overweight status, resulting in an average increase of 0.83 SDS more than controls. Subjects who were overweight at the outset experienced greater gains in both weight and stature SDS. Treatment had no statistically significant impact on weight-for-stature or BMI SDS. A reduction in body fat percentage was observed in those treated, both before (−6.1 %) and after (−4.3 %) adjustment for initial overweight status.


Early GH replacement has a positive effect on short-term statural outcomes in children with ONH and GHD, even in those exhibiting normal initial linear growth. Results were less conclusive regarding treatment effects on body composition and lipids.
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