title: Trends in steroid therapy for Duchenne muscular dystrophy in Japan.
abstract: We conducted a study to reveal trends in steroid prescription for Duchenne muscular dystrophy (DMD) patients in Japan.
  
  
  
  We asked patients (ages 5-20 years) identified in the patient registry and their clinicians about steroid therapy experiences. Regimen, dose, and starting age were compared among 3 subgroups according to prednisolone initiation year (2000-2004, 2005-2009, and 2010-2013).
  
  
  
  Among 157 prednisolone users, 4 different regimens were used. Dose frequencies were: every other day (98 patients), daily (44 patients), 10 days on 20 days off (14 patients), and weekly (1 patient). Median starting age was 6 years, and median dose was 0.42 mg/kg/day. There was an increase in daily regimen use from 2005-2009 (n = 9, 16%) to 2010-2013 (n = 33, 36%).
  
  
  
  This study revealed a transition over time in steroid use from expert opinion to evidence-based recommendation. Clinical research should be encouraged to optimize medication worldwide. Muscle Nerve 54: 673-680, 2016.
authors.affiliation: Department of Clinical Research Support, Translational Medical Center, National Center of Neurology and Psychiatry, Tokyo, Japan.
authors.forename: Fumi
authors.initials: F
authors.lastname: Takeuchi
authors.affiliation: Department of Child Neurology, National Center of Neurology and Psychiatry 4-1-1 Ogawa-higashi, Kodaira, Tokyo, 187-8551, Japan. komakih@ncnp.go.jp.
authors.forename: Hirofumi
authors.initials: H
authors.lastname: Komaki
authors.affiliation: Department of Clinical Research Support, Translational Medical Center, National Center of Neurology and Psychiatry, Tokyo, Japan.
authors.forename: Harumasa
authors.initials: H
authors.lastname: Nakamura
authors.affiliation: Department of Neuropsychopharmacology, National Institute of Mental Health, National Center of Neurology and Psychiatry, Tokyo, Japan.
authors.forename: Naohiro
authors.initials: N
authors.lastname: Yonemoto
authors.affiliation: Department of Clinical Epidemiology, Translational Medical Center, National Center of Neurology and Psychiatry, Tokyo, Japan.|Department of Biostatistics, School of Public Health, The University of Tokyo, Tokyo, Japan.
authors.forename: Kousuke
authors.initials: K
authors.lastname: Kashiwabara
authors.affiliation: Department of Clinical Research Support, Translational Medical Center, National Center of Neurology and Psychiatry, Tokyo, Japan.
authors.forename: En
authors.initials: E
authors.lastname: Kimura
authors.affiliation: Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan.
authors.forename: Shin'ichi
authors.initials: S
authors.lastname: Takeda
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D005260:Female; D005938:Glucocorticoids / Q000627:therapeutic use*; D006801:Humans; D007564:Japan / Q000453:epidemiology; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000175:diagnosis / Q000188:drug therapy* / Q000453:epidemiology*; D011239:Prednisolone / Q000627:therapeutic use*; D011795:Surveys and Questionnaires; D055815:Young Adult
chemical_list: D005938:Glucocorticoids; D011239:Prednisolone
keywords: Duchenne muscular dystrophy; clinical research; expert opinion; guideline; patient registry; regimen; steroid therapy