Prednisolone improves walking in Japanese Duchenne muscular dystrophy patients

详细信息    查看全文

• 作者：Fumi Takeuchi (1)
  Naohiro Yonemoto (2)
  Harumasa Nakamura (3)
  Reiko Shimizu (2) (4)
  Hirofumi Komaki (1) (2)
  Madoka Mori-Yoshimura (3)
  Yukiko K. Hayashi (2) (6)
  Ichizo Nishino (2)
  Mitsuru Kawai (5)
  En Kimura (2)
  Shin?chi Takeda (2)
  
• 关键词：Duchenne muscular dystrophy ; Prednisolone ; Walking ; National registry ; Natural history
• 刊名：Journal of Neurology
• 出版年：2013
• 出版时间：December 2013
• 年：2013
• 卷：260
• 期：12
• 页码：3023-3029
• 全文大小：
• 作者单位：Fumi Takeuchi (1)
  Naohiro Yonemoto (2)
  Harumasa Nakamura (3)
  Reiko Shimizu (2) (4)
  Hirofumi Komaki (1) (2)
  Madoka Mori-Yoshimura (3)
  Yukiko K. Hayashi (2) (6)
  Ichizo Nishino (2)
  Mitsuru Kawai (5)
  En Kimura (2)
  Shin?chi Takeda (2)
  
  1. Department of Child Neurology, National Centre Hospital, National Centre of Neurology and Psychiatry, 4-1-1 Ogawahigashi, Kodaira, Tokyo, 187-8551, Japan
  2. Translational Medical Centre, National Centre of Neurology and Psychiatry, 4-1-1, Ogawa-Higashi, Kodaira, Tokyo, 187-8551, Japan
  3. Department of Neurology, National Centre Hospital, National Centre of Neurology and Psychiatry, 4-1-1 Ogawahigashi, Kodaira, Tokyo, 187-8551, Japan
  4. Tokyo Women? Medical University, 8-1, Kawada-cho, Shinjuku-ku, Tokyo, 162-8666, Japan
  6. Department of Neurophysiology, Tokyo Medical University, 6-1-1 Shinjuku, Shinjuku-ku, Tokyo, 160-8402, Japan
  5. Higashi-Saitama National Hospital, 4147, Kurohama, Hasuda, Saitama, 349-0196, Japan
  
• ISSN：1432-1459

文摘

We evaluated the long-term efficacy of prednisolone (PSL) therapy for prolonging ambulation in Japanese patients with genetically confirmed Duchenne muscular dystrophy (DMD). There were clinical trials have shown a short-term positive effect of high-dose and daily PSL on ambulation, whereas a few study showed a long-term effect. Especially in Japan, ?eal-life?observation was lacking. We utilized the national registry of muscular dystrophy in Japan for our retrospective study. We compared the age at loss of ambulation (LOA) between patients in PSL group and those in without-PSL group. Out of 791 patients?in the Remudy DMD/BMD registry from July 2009 to June 2012, 560 were matched with inclusion criteria. Of the 560, all were genetically confirmed DMD patients, 245 (43.8?%) of whom were treated with PSL and 315 (56.2?%) without PSL. There was no difference between the two groups regarding their mutational profile. The age at LOA was significantly greater (11?month on average) in the PSL group than in the without-PSL group (median, 132 vs. 121?months; p?=?0.0002). Although strictly controlled clinical trials have shown that corticosteroid therapies achieved a marked improvement in ambulation, discontinuation of the drug due to intolerable side effects led to exclusion of clinical trial participants, which is considered as unavoidable. In our study, patients were not excluded from the PSL group, even if they discontinued the medication shortly after starting it. The results of our study may provide evidence to formulate recommendations and provide a basis for realistic expectations for PSL treatment of DMD patients in Japan, even there are certain limitations due to the retrospectively captured data in the registry.