Elsevier

Brain and Development

Volume 42, Issue 3, March 2020, Pages 277-288
Brain and Development

Original article
Clinical practice with steroid therapy for Duchenne muscular dystrophy: An expert survey in Asia and Oceania

https://doi.org/10.1016/j.braindev.2019.12.005Get rights and content
Under a Creative Commons license
open access

Abstract

Background

Several studies on clinical practice for Duchenne muscular dystrophy (DMD) have been conducted in Western countries. However, there have been only a few similar studies in Asia and Oceania. Here, we investigate the steroid therapy-related clinical practice for DMD among the local experts. In 2015, we conducted a DMD expert survey in Asia and Oceania to acquire information regarding patients with DMD and to assess current clinical practice with the cooperation of Asian and Oceanian Myology Centre, a neuromuscular disease research network.

Results

We obtained survey responses from 87 out of 148 clinicians (62%) from 13 countries and regions. In China, 1385 DMD patients were followed-up by 5 respondent neurologists, and 84% were between 0 and 9 years of age (15% were 10–19 years, 1% > 19 years). While in Japan, 1032 patients were followed-up by 20 clinicians, and the age distribution was similar between the 3 groups (27% were 0–9 years, 35% were 10–19 years, 38% were >19 years). Most respondent clinicians (91%) were aware of DMD standard of care recommendations. Daily prednisolone/prednisone administration was used most frequently at initiation (N = 45, 64%). Inconsistent opinion on steroid therapy after loss of ambulation and medication for bone protection was observed.

Conclusions

Rare disease research infrastructures have been underdeveloped in many of Asian and Oceanian countries. In this situation, our results show the snapshots of current medical situation and clinical practice in DMD. For further epidemiological studies, expansion of DMD registries is necessary.

Abbreviations

AOMC
Asian and Oceanian Myology Centre
BMD
Becker muscular dystrophy
DFZ
Deflazacort
DMD
Duchenne muscular dystrophy
DXA
Dual-energy X-ray absorptiometry
PSL
Prednisolone/prednisone
SAR
Strategic Administrative Region
TREAT-NMD
Translational Research in Europe – Assessment & Treatment of Neuromuscular Diseases

Keywords

Duchenne muscular dystrophy
Steroid therapy
Clinical practice
Epidemiology
Care recommendation
Asian and Oceanian Myology Center

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