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

Purpose To determine the prevalence and characteristics of eyes treated for retinopathy of prematurity (ROP) not meeting currently recommended early treatment (type 1) criteria. Methods This was a secondary analysis of data from the Postnatal Growth and ROP (G-ROP) Study, a retrospective cohort study of 7,483 infants undergoing ROP examinations and treatment at 29 North American hospitals between January 2006 and June 2012. Medical records were reviewed to determine the prevalence and characteristics of eyes treated for ROP less severe than type 1 ROP. Results Of 1,004 eyes that received ROP treatment, 126 eyes of 91 infants (0.8% of all eyes; 12.5% of treated eyes) underwent treatment for ROP less severe than type 1. Mean age at treatment was 38 weeks’ post-menstrual age (range, 32-49 weeks). Reasons for treatment included type 1 ROP in the fellow eye (43%), stage 3 ROP with pre-plus in the treated eye (30%), concerning structural changes in the retina (7%), persistent stage 3 ROP for ≥6 weeks without regression (6%), stage 3 ROP with no plus disease in the treated eye (5%), stage 3, zone III ROP with plus disease (3%), logistical considerations (3%), or stage 2 disease in the treated eye (2%). Conclusions Of all eyes treated for ROP, 1/8 were treated for disease less severe than currently recommended type 1 criteria. Clinician judgment of risk for permanent vision impairment superseded recommended treatment criteria and was usually related to type 1 disease in the fellow eye or pre-plus vascular changes in one or both eyes.

Purpose To determine whether a multivariate risk model can select infants with low-risk eyes for an alternative protocol that reduces retinopathy of prematurity (ROP) screening without loss of effectiveness. Methods This was a retrospective, nonrandomized, comparative study. We assigned 712 eyes of 357 premature infants of 401–1,250 g birth weights as high or low risk for prethreshold or threshold ROP using a risk model with variables of birth weight, gestational age, multiple birth, race, and gender. Using simulations, infants with high-risk eyes ( p ≥ 0.15) were screened conventionally, while those with low-risk eyes were screened with the 35q3 protocol (initial examination at 35 weeks postmenstrual age followed by screening every 3 weeks, with increased or decreased intervals based on ROP severity). The resultant reduction in ROP screening and the delay to detection of severe ROP were calculated. Results The 35q3 protocol reduced the mean number of eye examinations per infant in the overall population by 13.4% ( p = 0.0051). No eyes had a delay to the detection of threshold or type 1 zone 1 ROP. Of a total of 57 type 1 ROP eyes, 2 eyes with type 1 zone 2 ROP were delayed to detection by 1 week. As our study was done prior to the Early Treatment of ROP study, the 2 eyes were observed and regressed without progression to threshold ROP. Conclusions A risk-based alternative screening protocol increased the efficiency of ROP screening for infants of 401–1,250 g birth weight without an apparent loss of effectiveness. This investigational approach requires further validation by multicenter studies.