Trends in steroid therapy for Duchenne muscular dystrophy in Japan

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.