Utilization of Heart Failure Medications in Duchenne Muscular Dystrophy Patients with Left Ventricular Systolic Dysfunction

Abstract

Purpose Current guidelines for the management of left ventricular systolic dysfunction (LVSD) in Duchenne Muscular Dystrophy (DMD) recommend angiotensin converting enzyme (ACE) inhibitors (class IIa). Little is known about the historic use of HF medications in Duchenne Muscular Dystrophy (DMD) and LVSD. Methods We performed a retrospective cohort study of DMD patients followed at 17 centers across North America from January 1, 2005-December 31, 2015. LVSD was defined as an ejection fraction (EF) Results LVSD was present in 209/436 (47.9%) DMD males including 84 (19.3%) patients with LVSD on baseline echocardiogram with median age 14 (IQR 11-17). 125 (28.7%) patients developed LVSD during the study with a median age at diagnosis of 15 years (IQR 12-17). Of the 209 patients with LVSD, 48 (23.0%) were never on HF medications, 105 (50.2%), 44 (21.0%), 11 (5.3%) and 1 (0.5%) were on single, dual, triple and quad HF therapy respectively. HF medications prescribed during the study period for those with LVSD included angiotensin converting enzyme inhibitor/angiotensin receptor blocker (ACE/ARB) (134, 64.1%), aldosterone antagonist (17, 8.1%), beta blocker (55, 26.3%), and digoxin (12, 5.7%). 154 (73.7%) were not on heart failure medications at study entry (baseline visit) and first line therapies included ACE/ARB (77, 51.0%), beta blockers (32, 21.2%), aldosterone antagonist (2, 1.3%), and digoxin (6, 4.0%). Overall therapies for these 154 patients included ACE/ARB (84, 55.6%), beta blocker (46, 30.5%), aldosterone antagonist (8, 5.3%) and digoxin (8, 5.3%). Conclusion There is significant practice variability that is not consistent with current consensus guidelines in the utilization of HF medications in DMD patients with LVSD. Almost 65% of the cohort were started on an ACE/ARB, however 23% were never prescribed a HF medication. Further studies are needed within the DMD population to understand the efficacy and decision making related to the prescription of common HF medications.