Summary
This study analyzed cardiac medication use in 265 males with Duchenne Muscular Dystrophy (DMD) using data from the ACTION Dystrophinopathy Registry to assess current practices and adherence to consensus directed medical therapy (CDMT). CDMT includes a combination of ACE inhibitors (ACEi), angiotensin II receptor blockers (ARB), or angiotensin receptor-neprilysin inhibitors (ARNI), plus a beta-blocker (BB), and a mineralocorticoid receptor antagonist (MRA).
Key Findings
- Median patient age: 17.5 years; median follow-up: 11.5 months.
- 57.7% had decreased left ventricular (LV) systolic function at follow-up.
- 25.3% had moderate or severe dysfunction; among them, ~73% were on CDMT, with no significant improvement from enrollment (p = 0.92).
- Only ~25–28% of patients on CDMT reached target doses of medications.
- Use of ACEi/ARB/ARNI, BB, and MRA was high among those with systolic dysfunction, but target dosing was rare.
- For patients with normal LV function, use of prophylactic medications like ACEi and MRA was common.
- SGLT2 inhibitors, though not standard in DMD, showed increased use and are being explored for potential benefit.
- Conclusion
While most patients with DMD and cardiac dysfunction were prescribed medications consistent with CDMT, many did not receive optimal dosing. About 30% of those with significant dysfunction were not on full CDMT. This highlights the need for:
- Better strategies to optimize treatment,
- Improved understanding of medication impact,
- Further evaluation of barriers to CDMT adherence, especially as cardiac complications become a leading cause of death in DMD.
This ACTION study was available online in Pediatric Cardiology on June 20, 2025. Congratulations to the authors:
- Carol A. Wittlieb-Weber
- Brian F. Birnbaum
- Chesney D. Castleberry
- Tyler W. Cunningham
- Paul Esteso
- Katheryn E. Gambetta
- Emily A. Hayes
- Daphne T. Hsu
- Beth D. Kaufman
- Benjamin Kroslowitz
- Ashwin K. Lal
- Angela Lorts
- Hugo Martinez
- Deepa Mokshagundam
- Deipanjan Nandi
- John J. Parent
- Frank Raucci
- Nelia Soares
- Jonathan H. Soslow
- Renata Shih
- Svetlana Shugh
- Chet R. Villa
- Sarah J. Wilkens
- Bethany L. Wisotzkey
- Jennifer Conway
CITATION
Wittlieb-Weber, C.A., Birnbaum, B.F., Castleberry, C.D. et al. Cardiac Medication Use in ACTION for Duchenne Muscular Dystrophy Cardiomyopathy. Pediatric Cardiology (2025). https://link.springer.com/article/10.1007/s00246-025-03917-2
