Growth hormone treatment in boys with Duchenne muscular dystrophy and glucocorticoid-induced growth failure.

Abstract

This study evaluated efficacy and safety of growth hormone treatment in Duchenne muscular dystrophy boys with glucocorticoid-induced growth failure. We reviewed 39 consecutive boys (average age 11.5 years; 32 ambulatory) treated with growth hormone for 1 year during a four-year period. Boys were on long-term daily deflazacort or prednisone (mean duration 5 ± 2.2 years; dosing regimen prednisone 0.75 mg/kg/day equivalent). Primary outcomes were growth velocity and height-for-age z-scores (height SD) at 1 year. Height velocity increased from 1.3 ± 0.2 to 5.2 ± 0.4 cm/year on growth hormone (p<0.0001). Pre-growth hormone decline in height SD (-0.5 ± 0.2SD/year) stabilized at height SD -2.9 ± 0.2 on growth hormone (p<0.0001). The rate of weight gain was unchanged, at 2.8 ± 0.6 kg/year pre-growth hormone and 2.6 ± 0.7 kg/year at 1 year. Motor function decline was similar pre-growth hormone and at 1 year. Cardiopulmonary function was unchanged. Three experienced side effects. In this first comprehensive report of growth hormone in Duchenne muscular dystrophy, growth hormone improved growth at 1 year, without detrimental effects observed on neuromuscular and cardiopulmonary function.

• Authors: Meilan M Rutter, James Collins, Susan R Rose, Jessica G Woo, Heidi Sucharew, Hemant Sawnani, Kan N Hor, Linda H Cripe, Brenda L Wong
• Journal: Neuromuscular disorders : NMD (Neuromuscul Disord) Vol. 22 Issue 12 Pg. 1046-56 (Dec 2012) ISSN: 1873-2364 [Electronic] England
• PMID: 22967789 (Publication Type: Journal Article)
• Copyright: Copyright © 2012 Elsevier B.V. All rights reserved.

Chemical References

• Glucocorticoids
• Pregnenediones
• Human Growth Hormone
• deflazacort
• Prednisone

Topics

• Body Height (drug effects)
• Body Weight (drug effects)
• Child
• Glucocorticoids (adverse effects, therapeutic use)
• Growth Disorders (chemically induced)
• Human Growth Hormone (adverse effects, therapeutic use)
• Humans
• Male
• Muscular Dystrophy, Duchenne (drug therapy, physiopathology)
• Prednisone (adverse effects, therapeutic use)
• Pregnenediones (adverse effects, therapeutic use)
• Treatment Outcome