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Published on: April 10, 2019
Trends in steroid therapy for Duchenne muscular dystrophy in Japan
Fumi Takeuchi1, Hirofumi Komaki2, Harumasa Nakamura1
1Department of Clinical Research Support, Translational Medical Center, National Center of Neurology and Psychiatry, Tokyo, 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.
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