Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy

 

Glucocorticoid treatment is recommended as a standard of care in Duchenne muscular dystrophy; however, few studies have assessed the long-term benefits of this treatment. In this prospective cohort study, the authors examined the long-term effects of glucocorticoids on milestone-related disease progression across the lifespan and survival in male patients aged 2-28 years with Duchenne muscular dystrophy at 20 centres in nine countries. Patients were followed up for 10 years. They compared no glucocorticoid treatment or cumulative treatment duration of less than 1 month versus treatment of 1 year or longer with regard to progression of nine disease-related and clinically meaningful mobility and upper limb milestones. They used Kaplan-Meier analyses to compare glucocorticoid treatment groups for time to stand from supine of 5 s or longer and 10 s or longer, and loss of stand from supine, four-stair climb, ambulation, full overhead reach, hand-to-mouth function, and hand function. Risk of death was also assessed. Time to all disease progression milestone events was significantly longer in patients treated with glucocorticoids for 1 year or longer than in patients treated for less than 1 month or never treated (log-rank p<0•0001). Glucocorticoid treatment for 1 year or longer was associated with increased median age at loss of mobility milestones by 2•1-4•4 years and upper limb milestones by 2•8-8•0 years compared with treatment for less than 1 month. Deflazacort was associated with increased median age at loss of three milestones by 2•1-2•7 years in comparison with prednisone or prednisolone (log-rank p<0•012). 45 patients died during the 10-year follow-up. 39 (87%) of these deaths were attributable to Duchenne-related causes in patients with known duration of glucocorticoids usage. 28 (9%) deaths occurred in 311 patients treated with glucocorticoids for 1 year or longer compared with 11 (19%) deaths in 58 patients with no history of glucocorticoid use (odds ratio 0•47, 95% CI 0•22-1•00; p=0•0501).

McDonald CM, Henricson EK, Abresch RTet al. Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study. Lancet. 2017 Nov 22. pii: S0140-6736(17)32160-8.