Therapeutic Options to Improve Bone Health Outcomes in Duchenne Muscular Dystrophy: Zoledronic Acid and Pubertal Induction | DuchenneXchange

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Scientific Articles

Therapeutic Options to Improve Bone Health Outcomes in Duchenne Muscular Dystrophy: Zoledronic Acid and Pubertal Induction

key information

source: Journal of Paediatrics and Child Health

year: 2017

authors: Angelina Lim, Margaret Zacharin, Janne Pitkin, Katy de Valle, Monique M Ryan, Peter J Simm

summary/abstract:

We report outcomes of nine corticosteroid‐dependent boys with Duchenne muscular dystrophy (DMD), aged =<18 years, treated in the last 10 years at The Royal Children’s Hospital (RCH), Melbourne, with at least one dose of zoledronic acid (ZA) for vertebral crush fracture (VCF) (defined using Genant criteria, with a loss of >25% anterior and/or middle and/or posterior vertebral height) and with pubertal induction where indicated. Long‐term oral corticosteroid therapy used in DMD causes inevitable, progressive bone loss and increased fracture risk with 20–50% of affected boys ambulant prior to fracture losing ability to walk after the fracture. Limited data exist on use of ZA in DMD, with only one published study to date examining its efficacy in vertebral fractures in DMD.

organisation: University of Melbourne, Australia

DOI: 10.1111/jpc.13692

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