Can deflazacort improve survival time?

Deflazacort improves survival time

Deflazacort is a glucocorticoid commonly used for anti-inflammatory and immunosuppressive purposes. Studies have shown that the vast majority of DMD patients without hormone therapy will lose mobility before the age of 12, while 70% to 80% of patients using deflazacort are still able to walk at this age. Treatment reduces the occurrence and severity of scoliosis and protects heart and lung function. The protection of the patient's heart is an important factor in extending the patient's life.

Deflazacort Reduces Risk of Death Trial

In a prospective cohort study, we enrolled men aged 2 to 28 years with Duchenne muscular dystrophy at 20 centers in nine countries. Followed up for 10 years. We compared progression of nine disease-related, clinically significant mobility and upper extremity milestones, comparing no treatment with glucocorticoids or cumulative treatment for less than 1 month with treatment for 1 year or more.

A total of 440 patients were recruited during the two recruitment periods. Patients who were treated with glucocorticoids for 1 year or more had significantly longer time to all disease progression milestones than patients who were treated with glucocorticoids for less than 1 month or who were never treated. Compared with patients treated for less than 1 month, patients treated with glucocorticoids for 1 year or longer had a median age increase of 2·1–4·4 years at loss of mobility milestones and 2·8–8·0 years at upper limb milestones.

Compared with prednisone or prednisolone, norcantharidin was associated with an increase in median age at loss of three milestones of 2·1–2·7 years. Forty-five patients died during the 10-year follow-up period. Thirty-nine (87%) deaths were attributed to Duchenne muscular dystrophy-related causes in patients with known duration of glucocorticoid use. Twenty-eight of 311 patients (9%) who had been treated with glucocorticoids for 1 year or more died, compared with 11 of 58 patients (19%) who had no history of glucocorticoid use (odds ratio 0.47).

Interpretation: In patients with Duchenne muscular dystrophy, treatment with glucocorticoids (eg, deflazacort) reduces the risk of clinically significant loss of mobility and disease progression milestones in the upper extremities and reduces the risk of death.

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References

McDonald CM, Henricson EK, Abresch RT, Duong T, Joyce NC, Hu F, Clemens PR, Hoffman EP, Cnaan A, Gordish-Dressman H; CINRG Investigators. Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study. Lancet. 2018 Feb 3;391(10119):451-461. doi: 10.1016/S0140-6736(17)32160-8. Epub 2017 Nov 22. PMID: 29174484.