Left ventricular dysfunction (LVD) is a uniform complication of childhood-onset dystrophinopathy. Historically, cardiac management began at LVD diagnosis by echocardiogram. Currently, prophylactic cardiac medication treatment is recommended. Diagnostic and treatment data collected by a multisite population-based surveillance program were analyzed to test associations between prophylactic cardiac medication and LVD onset and all-cause mortality. Medication use (cardiac, corticosteroids) and echocardiogram results were analyzed for 685 males. LVD was defined as an ejection fraction below 55% or, if missing, shortening fraction below 28%. Onset age was the midpoint between ages at last normal and first abnormal measurement. Cardiac treatment was considered prophylactic if a cardiac medication was initiated the year before, or in the absence of, LVD. Age at death was determined from medical records or death certificates. Analytic methods included Kaplan-Meier curve comparisons and Cox proportional hazards modeling to estimate hazards ratios (HR)s with 95% confidence intervals (CI). LVD was diagnosed for 32% (mean onset age 11.9 years) and prophylactic cardiac medication was identified for 16% of all males. An angiotensin-converting enzyme inhibitor was the most common prescribed class of prophylactic cardiac medication (79.2%). Greater risk of LVD was found among those not receiving prophylactic treatment compared to those who did (HR = 3.2, 95%CI = 2.0,5.0). Prophylactic cardiac medication plus corticosteroids was associated with longest survival without LVD (18.8 years) compared to all other medication combinations (range: 16.2-16.8 years; log-rank = 29.5, p < .0001). Hazard of death was 2 times higher (crude HR) among those not receiving prophylactic cardiac medication compared to those who did (HR = 2.1, 95%CI = 1.0,4.1). Our findings suggest prophylactic cardiac medication delays LVD onset and may reduce risk of death in childhood-onset dystrophinopathy.
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