Improved Outcomes in Duchenne Muscular Dystrophy With Long-Term Glucocorticoid Treatment

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Patients who received deflazacort were older when they reached disease milestones compared with patients taking prednisolone or prednisone.

Lifelong use of glucocorticoids is associated with physical strength and function improvements, greater health-related quality of life, and improved survival in patients with Duchenne muscular dystrophy, according to findings from a prospective cohort study published in the Lancet.

Investigators enrolled 440 male patients with Duchenne muscular dystrophy from a total of 20 health centers during 2006-2009 and 2012-2016. Treatment with glucocorticoids for <1 month was compared with glucocorticoid treatment of ≥1 year, with researchers evaluating the differences between the 2 groups in regard to progression of clinically meaningful mobility and upper limb milestones.

Among participants managed with glucocorticoids for ≥1 year, time to all disease progression milestones was longer than for those treated <1 month or those never treated with the therapy (P <.0001).

Compared with treatment <1 month, patients managed with glucocorticoids for ≥1 year were older at loss of upper limb milestones by 2.8 to 8.0 years and at loss of mobility milestones by 2.1 to 4.4 years. In addition, the use of deflazacort was associated with a higher median age at loss of 3 milestones by 2.1 to 2.7 years when compared with treatment with prednisolone or prednisone (P <.012).

Of the 45 patients who died during the 10-year follow-up period, mortality related to Duchenne muscular dystrophy occurred in 39 patients who were using glucocorticoids. A significantly higher proportion of deaths occurred among patients who never used glucocorticoids vs those who used the therapy for ≥1 year (19% vs 9%, respectively; odds ratio 0.47, 95% CI, 0.22-1.00; P =.0501).

The “emerging changes in care standards” among steroid-treated vs nontreated patients was cited by the investigators of this study as a potential limitation that resulted in strong imbalances between the groups. Also, the researchers did not perform a multivariate analysis for mortality, which may further limit the findings.

The effect glucocorticoid use had on survival in this study “is likely to be caused by a combination of positive effects on pulmonary musculature (preserving airway clearance and ventilation) and cardiomyopathy,” the authors noted.


McDonald CM, Henricson EK, Abresch RT, et al. Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study [published online November 22, 2017]. Lancet. doi:10.1016/S0140-6736(17)32160-8