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

Takeuchi F 1 , Nakamura H 2 , Yonemoto N 3 , Komaki H 4 , Rosales RL 5 , Kornberg AJ 6 Show all authors , Bretag AH 7 , Dejthevaporn C 8 , Goh KJ 9 , Jong YJ 10 , Kim DS 11 , Khadilkar SV 12 , Shen D 13 , Wong KT 14 , Chai J 15 , Chan SH 16 , Khan S 17 , Ohnmar O 18 , Nishino I 19 , Takeda S 20 , Nonaka I 21

Affiliations 

  • 1 Department of Clinical Research Support, Translational Medical Center, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-higashi, Kodaira, Tokyo 187-8551, Japan
  • 2 Department of Clinical Research Support, Translational Medical Center, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-higashi, Kodaira, Tokyo 187-8551, Japan. Electronic address: [email protected]
  • 3 Department of Biostatistics, Kyoto University School of Public Health, Yoshidakonoe, Sakyo, Kyoto, Kyoto 606-8501, Japan
  • 4 Department of Clinical Research Support, Translational Medical Center, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-higashi, Kodaira, Tokyo 187-8551, Japan; Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-higashi, Kodaira, Tokyo 187-8551, Japan
  • 5 The Department of Neurology & Psychiatry and The NeuroScience Institute, University of Santo Tomas Hospital, España Blvd, Sampaloc, Manila, 1008 Metro Manila, Philippines
  • 6 Children's Neuroscience Centre, The Royal Children's Hospital Melbourne, 50 Flemington Road, Parkville, Victoria 3052, Melbourne, Australia
  • 7 School of Pharmacy and Medical Sciences, University of South Australia, Adelaide, SA 5001, Australia
  • 8 Department of Medicine, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok 10400, Thailand
  • 9 Division of Neurology, Department of Medicine, Faculty of Medicine, University of Malaya, 50603 Kuala Lumpur, Malaysia
  • 10 Departments of Pediatrics and Laboratory Medicine, Kaohsiung Medical University Hospital/Graduate Institute of Clinical Medicine, College of Medicine, Kaohsiung Medical University, Taiwan 100, Shih-Chuan 1st Rd, Kaohsiung 80708, Taiwan; College of Biological Science and Technology, National Chiao Tung University, 75 Po-Ai St, Hsinchu 30068, Taiwan
  • 11 Department of Neurology, Pusan National University Yangsan Hospital, 20 Kumo-ro, Yangsan, Gyeongnam, Republic of Korea
  • 12 Department of Neurology, Bombay Hospital Institute of Medical Sciences, 12, Marine Lines, Mumbai, Maharashtra 40020, India
  • 13 Fudan University, 220 Handan Rd, WuJiaoChang, Yangpu District, Shanghai 200433, China
  • 14 Department of Pathology, Faculty of Medicine, University of Malaya, 50603 Kuala Lumpur, Malaysia
  • 15 Department of Neurology, National Neuroscience Institute, 11 Jalan Tan Tock Seng, Singapore 308433, Singapore
  • 16 Division of Paediatric Neurology, Department of Paediatrics and Adolescent Medicine, Queen Mary Hospital, The University of Hong Kong, Hong Kong Special Administrative Region
  • 17 Department of Neurology, Aga Khan University Hospital, Stadium Rd, Karachi, Karachi City, Sindh 74800, Pakistan
  • 18 Department of Medical Neurology, University of Medicine 1, No. 245, Myoma Kyaung Street, Lanmadaw Township, Yangon, Myanmar
  • 19 Department of Neuromuscular Research, National Institute of Neurosciences, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-higashi, Kodaira, Tokyo 187-8551, Japan
  • 20 Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-higashi, Kodaira, Tokyo 187-8551, Japan
  • 21 Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-higashi, Kodaira, Tokyo 187-8551, Japan; Department of Neuromuscular Research, National Institute of Neurosciences, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-higashi, Kodaira, Tokyo 187-8551, Japan
Brain Dev, 2020 Mar;42(3):277-288.
PMID: 31980267 DOI: 10.1016/j.braindev.2019.12.005

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.

* Title and MeSH Headings from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.

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