title: 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.affiliation: Division of Endocrinology, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, OH 45229, USA. meilan.rutter@cchmc.org
authors.forename: Meilan M
authors.initials: MM
authors.lastname: Rutter
authors.forename: James
authors.initials: J
authors.lastname: Collins
authors.forename: Susan R
authors.initials: SR
authors.lastname: Rose
authors.forename: Jessica G
authors.initials: JG
authors.lastname: Woo
authors.forename: Heidi
authors.initials: H
authors.lastname: Sucharew
authors.forename: Hemant
authors.initials: H
authors.lastname: Sawnani
authors.forename: Kan N
authors.initials: KN
authors.lastname: Hor
authors.forename: Linda H
authors.initials: LH
authors.lastname: Cripe
authors.forename: Brenda L
authors.initials: BL
authors.lastname: Wong
mesh_terms: D001827:Body Height / Q000187:drug effects; D001835:Body Weight / Q000187:drug effects; D002648:Child; D005938:Glucocorticoids / Q000009:adverse effects* / Q000627:therapeutic use; D006130:Growth Disorders / Q000139:chemically induced*; D019382:Human Growth Hormone / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000503:physiopathology; D011241:Prednisone / Q000009:adverse effects / Q000627:therapeutic use; D011282:Pregnenediones / Q000009:adverse effects* / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D005938:Glucocorticoids; D011282:Pregnenediones; D019382:Human Growth Hormone; C021988:deflazacort; D011241:Prednisone