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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...

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Detalles Bibliográficos
Autores principales: Takeuchi, Fumi, Komaki, Hirofumi, Nakamura, Harumasa, Yonemoto, Naohiro, Kashiwabara, Kousuke, Kimura, En, Takeda, Shin'ichi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5113709/
https://www.ncbi.nlm.nih.gov/pubmed/26910583
http://dx.doi.org/10.1002/mus.25083
Descripción
Sumario: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