(51) Taking ACTION. Creation of a Prospective Registry of Boys with Dystrophinopathy and Ventricular Dysfunction to Define Cardiac Medication Use and Optimize Guideline Directed Medical Therapy

• Published in: The Journal of heart and lung transplantation Vol. 42; no. 4; p. S32
• Main Authors: Wittlieb-Weber, C.A., Birnbaum, B., Castleberry, C., Esteso, P., Gambetta, K., Hayes, E., Hsu, D., Kaufman, B., Lal, A., Lorts, A., Martinez, H., Mokshagundam, D., Nandi, D., Parent, J., Raucci, F., Soares, N., Shezad, M., Shih, R., Shugh, S., Villa, C., Wilkens, S., Wisotzkey, B.L., Conway, J.
• Format: Journal Article
• Language: English
• Published: Elsevier Inc 01.04.2023
• ISSN: 1053-2498; 1557-3117
• DOI: 10.1016/j.healun.2023.02.067

This project seeks to define cardiac medication use for boys with Duchenne and Becker Muscular Dystrophy (DBMD) and ventricular dysfunction to optimize the use of guideline directed medical therapy (GDMT). DBMD subjects with a history of left ventricular ejection fraction (EF) ≤ 45% were enrolled in an Advanced Cardiac Therapies Improving Outcomes Network (ACTION) database. Cardiac medication use in the presence of dysfunction was analyzed at enrollment. GDMT was defined as current use of angiotensin-converting-enzyme inhibitor/angiotensin II receptor blocker /angiotensin receptor-neprilysin inhibitor plus beta-blocker plus mineralocorticoid receptor antagonist. 128 boys with DBMD from 20 centers in North America were enrolled; median age of 18.9 [IQR 16.8-21.9] years with 29 (22.7%) ambulatory, 61 (47.7%) using respiratory support, and 59 (46.1%) on steroids. The median EF recorded (N=107, 84%) at enrollment was 41.8% [IQR 34%-49.5%]. Cardiac medication use is outlined in Table 1. Frequency of use of GDMT by EF is shown in Figure 1. GDMT was being used for 79 (43.4%) subjects at the time of enrollment; 8/15 (53.3%) of those with severe ventricular dysfunction (EF <30%) were on GDMT. Initial analysis of a prospective registry of boys with DBMD and ventricular dysfunction shows that the majority are not receiving GDMT. More work is needed to better understand barriers to optimization of GDMT particularly given the extension of life for DMD patients and increasing frequency of cardiac causes of death.