Clinical practice with steroid therapy for Duchenne muscular dystrophy: An expert survey in Asia and Oceania.
Adolescent
Adult
Child
Child, Preschool
China
Health Care Surveys
Humans
Infant
Japan
Male
Muscular Dystrophy, Duchenne
/ drug therapy
Oceania
Practice Patterns, Physicians'
/ statistics & numerical data
Prednisolone
/ therapeutic use
Prednisone
/ therapeutic use
Societies, Medical
/ statistics & numerical data
Steroids
/ therapeutic use
Young Adult
Asian and Oceanian Myology Center
Care recommendation
Clinical practice
Duchenne muscular dystrophy
Epidemiology
Steroid therapy
Journal
Brain & development
ISSN: 1872-7131
Titre abrégé: Brain Dev
Pays: Netherlands
ID NLM: 7909235
Informations de publication
Date de publication:
Mar 2020
Mar 2020
Historique:
received:
07
07
2019
revised:
04
12
2019
accepted:
06
12
2019
pubmed:
26
1
2020
medline:
24
10
2020
entrez:
26
1
2020
Statut:
ppublish
Résumé
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. 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. 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.
Sections du résumé
BACKGROUND
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
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
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.
Identifiants
pubmed: 31980267
pii: S0387-7604(19)30713-2
doi: 10.1016/j.braindev.2019.12.005
pii:
doi:
Substances chimiques
Steroids
0
Prednisolone
9PHQ9Y1OLM
Prednisone
VB0R961HZT
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
277-288Informations de copyright
Copyright © 2020 The Japanese Society of Child Neurology. Published by Elsevier B.V. All rights reserved.