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

Issued Date

2020-03-01

Resource Type

Article

ISSN

18727131
03877604

DOI

10.1016/j.braindev.2019.12.005

Other identifier(s)

2-s2.0-85078191407

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Mahidol University

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SCOPUS

Bibliographic Citation

Brain and Development. Vol.42, No.3 (2020), 277-288

Suggested Citation

Fumi Takeuchi, Harumasa Nakamura, Naohiro Yonemoto, Hirofumi Komaki, Raymond L. Rosales, Andrew J. Kornberg, Allan H. Bretag, Charungthai Dejthevaporn, Khean Jin Goh, Yuh Jyh Jong, Dae Seong Kim, Satish V. Khadilkar, Dingguo Shen, Kum Thong Wong, Josiah Chai, Sophelia Hoi Shan Chan, Sara Khan, Ohnmar Ohnmar, Ichizo Nishino, Shin'ichi Takeda, Ikuya Nonaka Clinical practice with steroid therapy for Duchenne muscular dystrophy: An expert survey in Asia and Oceania. Brain and Development. Vol.42, No.3 (2020), 277-288. doi:10.1016/j.braindev.2019.12.005 Retrieved from: https://repository.li.mahidol.ac.th/handle/20.500.14594/53747

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Title

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

Author(s)

Fumi Takeuchi
 Harumasa Nakamura
 Naohiro Yonemoto
 Hirofumi Komaki
 Raymond L. Rosales
 Andrew J. Kornberg
 Allan H. Bretag
 Charungthai Dejthevaporn
 Khean Jin Goh
 Yuh Jyh Jong
 Dae Seong Kim
 Satish V. Khadilkar
 Dingguo Shen
 Kum Thong Wong
 Josiah Chai
 Sophelia Hoi Shan Chan
 Sara Khan
 Ohnmar Ohnmar
 Ichizo Nishino
 Shin'ichi Takeda
 Ikuya Nonaka

Other Contributor(s)

Kyoto University School of Public Health
 Kaohsiung Medical University Chung-Ho Memorial Hospital
 University of Santo Tomas Hospital
 The Aga Khan University Hospital
 University of South Australia
 University of Malaya
 Royal Children's Hospital, Melbourne
 National Neuroscience Institute of Singapore
 National Institute of Neuroscience, Kodaira
 National Center of Neurology and Psychiatry Kodaira
 Faculty of Medicine, Ramathibodi Hospital, Mahidol University
 National Chiao Tung University Taiwan
 Bombay Hospital and Medical Research Centre
 Fudan University
 The University of Hong Kong
 Pusan National University Yangsan Hospital
 University of Medicine 1

Abstract

© 2020 The Japanese Society of Child Neurology 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.

Keyword(s)

Medicine
 Neuroscience

Availability

URI

https://repository.li.mahidol.ac.th/handle/20.500.14594/53747

Collections

Scopus 2020