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Abstracts & Posters

Testosterone therapy in patients with Duchenne muscular dystrophy and glucocorticoid-induced pubertal delay

key information

source: Neuromuscular Disorders

year: 2017

authors: C. Keefe, B. Wong, I. Rybalsky, K. Shellenbarger, C. Tian, J. Khoury, L. Hornung, M. Rutter


Glucocorticoid therapy slows disease progression in Duchenne muscular dystrophy (DMD), but causes significant adverse effects, including delayed puberty or hypogonadism, obesity, insulin resistance and osteoporosis. Lack of puberty or hypogonadism further increases risk for osteoporosis and affects quality of life, but is not consistently addressed in DMD care. We sought to determine if testosterone therapy could induce puberty, and to examine the effects of testosterone therapy on body composition, bone health and metabolic indices, in glucocorticoid-treated patients with DMD. We reviewed medical records of boys with DMD on long-term glucocorticoid therapy, who were treated with testosterone for delayed (n = 31) or arrested (n = 2) puberty. Outcomes of interest were endogenous pubertal development; changes in body composition; bone mineral density and content of lumbar spine, whole body and lateral distal femur by dual-energy X-ray absorptiometry; and metabolic laboratory indices. Changes in bone health measures within 2.5 years prior to treatment were compared to changes up to 2.5 years after treatment initiation. Thirty-three male patients (median age 15.0 years, 27% ambulatory) on glucocorticoid therapy (median duration 7.7 years) were treated with testosterone therapy for a median of 37 (range 4.1 to 73.5) months. Endogenous puberty developed in 45% of previously pre-pubertal boys after starting testosterone. We observed increases in spine and whole body absolute bone mineral content (p = 0.03 and 0.0002 respectively), and stabilization of fat mass and percentage of total body fat accrual, when comparing post- to pretestosterone treatment initiation. Although longer-term data are needed, testosterone therapy should be considered in the management of glucocorticoid treated male patients with DMD who experience delayed puberty.

organization: Cincinnati Children's Hospital Medical Center, USA

DOI: 10.1016/j.nmd.2017.06.044

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