Real-world outcomes of long-term prednisone and deflazacort use in patients with Duchenne muscular dystrophy: experience at a single, large care center

• Published in: Journal of comparative effectiveness research 2020-02, Vol.9 (3), p.177-189
• Main Authors: Marden, Jessica R, Freimark, Jonathan, Yao, Zhiwen, Signorovitch, James, Tian, Cuixia, Wong, Brenda L
• Format: Article
• Language: English
• Subjects: Adolescent; Age; Bone Density; Cardiac function; Child; Child, Preschool; deflazacort; Duchenne muscular dystrophy; Electronic health records; FDA approval; Humans; Male; Muscular dystrophy; Muscular Dystrophy, Duchenne - drug therapy; Muscular Dystrophy, Duchenne - physiopathology; Patients; prednisone; Prednisone - therapeutic use; Pregnenediones - therapeutic use; real-world data; Retrospective Studies; Scoliosis; Steroids; Stroke Volume; Ventricular Function, Left; Walking
• ISSN: 2042-6305; 2042-6313
• DOI: 10.2217/cer-2019-0170

To assess outcomes among patients with Duchenne muscular dystrophy receiving deflazacort or prednisone in real-world practice. Clinical data for 435 boys with Duchenne muscular dystrophy from Cincinnati Children’s Hospital Medical Center were studied retrospectively using time-to-event and regression analyses. Median ages at loss of ambulation were 15.6 and 13.5 years among deflazacort- and prednisone-initiated patients, respectively. Deflazacort was also associated with a lower risk of scoliosis and better ambulatory function, greater % lean body mass, shorter stature and lower weight, after adjusting for age and steroid duration. No differences were observed in whole body bone mineral density or left ventricular ejection fraction. This single center study adds to the real-world evidence associating deflazacort with improved clinical outcomes. This retrospective study described outcomes for boys with Duchenne muscular dystrophy treated at Cincinnati Children’s Hospital Medical Center with the glucocorticoids deflazacort (∼95% daily dosing) or prednisone (∼68% daily dosing). Patients receiving deflazacort had lower risk of losing ambulation, lost ambulation at older ages than those receiving prednisone, and had lower risk of scoliosis. Across clinic visits, deflazacort was associated with greater preservation of ambulatory and pulmonary function, shorter stature, lower bodyweight and greater % lean body mass. This study adds to the evidence associating real-world dosing of deflazacort with improved outcomes for patients with Duchenne muscular dystrophy.