Cataract development associated with long-term glucocorticoid therapy in Duchenne muscular dystrophy patients

To evaluate the development of cataracts or elevated intraocular pressure (IOP) in patients with Duchenne muscular dystrophy (DMD) on long-term glucocorticoid (GC) treatment.

The medical records of DMD patients evaluated from 2010 to 2015 at a single center were reviewed retrospectively. The main outcome measures were prevalence of cataracts and elevated IOP, age of first detection of cataract, time from initial steroid use to first detection of cataract, and relative risk of cataract development for deflazacort versus prednisone treatment.

Of 596 DMD patients, 514 underwent GC therapy; all but one was male. The racial distribution was 82.1% white, 1.0% African American, 5.0% Hispanic, 2.9% Asian, and 8.0% more than one race or "other." The prevalence of cataracts was 22.4% in patients on GC therapy. The mean age at which cataract formation was first documented was 12.9 ± 4.1 years (IQR, 9.6-14.6). The mean time from initial steroid use to the first detection of cataract was 6.5 ± 3.6 years (IQR, 4.0-8.6). The odds of cataract development were 2.4-fold higher for patients on deflazacort compared with prednisone (95% CI, 1.3-4.5; P = 0.004). Only 7 patients (1.4%) underwent cataract surgery, at a mean age of 16.9 years (range, 10.7-24.6 years); all were on deflazacort. Among patients with available intraocular pressure measurements, elevated IOP occurred in only 1 patient (1.1%), who was on deflazacort.

In patients undergoing GC therapy for DMD, the rate of cataract formation was slow and well tolerated, with a higher risk among deflazacort patients. The percentage of patients requiring cataract extraction or with elevated IOP was very small. These findings suggest that a schedule of annual eye examinations is appropriate.