Trends in steroid therapy for Duchenne muscular dystrophy in Japan

• Published in: Muscle & nerve 2016-10, Vol.54 (4), p.673-680
• Main Authors: Takeuchi, Fumi, Komaki, Hirofumi, Nakamura, Harumasa, Yonemoto, Naohiro, Kashiwabara, Kousuke, Kimura, En, Takeda, Shin'ichi
• Format: Article
• Language: English
• Subjects: Adolescent; Child; Child, Preschool; Clinical Research; Cohort Studies; Duchenne muscular dystrophy; expert opinion; Female; Glucocorticoids - therapeutic use; guideline; Humans; Japan - epidemiology; Male; Muscular dystrophy; Muscular Dystrophy, Duchenne - diagnosis; Muscular Dystrophy, Duchenne - drug therapy; Muscular Dystrophy, Duchenne - epidemiology; patient registry; Patients; Prednisolone - therapeutic use; regimen; steroid therapy; Surveys and Questionnaires; Trends; Young Adult
• ISSN: 0148-639X; 1097-4598
• DOI: 10.1002/mus.25083

ABSTRACT Introduction: We conducted a study to reveal trends in steroid prescription for Duchenne muscular dystrophy (DMD) patients in Japan. Methods: 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). Results: 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%). Conclusions: 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