McDonald CM, et al. - This investigation was conducted to determine the long-term impacts of glucocorticoids on milestone-related disease progression across the lifespan and survival in patients with Duchenne muscular dystrophy. As a result of treating Duchenne muscular dystrophy patients with glucocorticoids, reduced risk of losing clinically meaningful mobility and upper limb disease progression milestones across the lifespan, as well as reduced risk of death, were reported.

Methods

• This prospective cohort study was performed at 20 centres in 9 countries including male patients aged 2–28 years with Duchenne muscular dystrophy.
• A 10-year follow-up was performed.
• With regard to progression of nine disease-related and clinically meaningful mobility and upper limb milestones, researchers compared no glucocorticoid treatment or cumulative treatment duration of less than 1 month vs treatment of 1 year or longer.
• A comparison was performed, using Kaplan-Meier analyses, for 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.
• In addition, researchers evaluated risk of death.

Results

• During two recruitment periods (2006–09 and 2012–16), a total of 440 patients were enrolled.
• Researchers found that in patients treated with glucocorticoids for 1 year or longer vs in patients treated for less than 1 month or never treated (log-rank p<0·0001), time to all disease progression milestone events was significantly longer.
• Data reported that compared with treatment for less than 1 month, 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.
• Results also revealed that in comparison with prednisone or prednisolone, deflazacort was related to increased median age at loss of three milestones by 2·1–2·7 years (log-rank p<0·012).
• During follow-up, death of 45 patients was reported.
• In patients with known duration of glucocorticoids usage, Duchenne-related causes were responsible for 39 (87%) of these deaths.
• In addition, occurrence of 28 (9%) deaths was reported in 311 patients treated with glucocorticoids for 1 year or longer vs 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).