Prophylactic Cardiac Medication May Prolong Survival in DMD

Prophylactic left ventricular dysfunction (LVD) treatment was associated with longer survival among males with Duchenne muscular dystrophy (DMD) in a study published in Muscle & Nerve.¹ However, only one-quarter of individuals received treatment, highlighting an area of DMD care where there is room for improvement, according to the study authors.

“Current care, including corticosteroids and management of respiratory compromise, has contributed to longer survival among those diagnosed with Duchenne muscular dystrophy (DMD); however, left ventricular dysfunction (LVD) continues to be a significant contributor to mortality,” the authors wrote. “Although prophylactic treatment with cardiac medications has been shown to delay the onset of LVD, epidemiological investigation into the association between prophylaxis and prolonged survival is limited.”

Prophylactic cardiac treatment was associated with prolonged survival in patients with Duchenne muscular dystrophy when adjusting for age at first visit and MD STARnet site. | Image credit: inthasone - stock.adobe.com

The retrospective study utilized data from the Muscular Dystrophy Surveillance, Tracking and Research Network (MD STARnet), a CDC-funded program that collects health information on patients with muscular dystrophy in certain US states.² The program is intended to help improve muscular dystrophy care and quality of life for patients. Following exclusions, the new study included 325 patients with DMD, with 94.5% classified as definite DMD.¹ Patients were at least 6 years old at the time of their last visit and were born between 1982 and 2009. Vital record linkages and medical record reviews determined death status.

A total of 90 patients (27.7%) had documented prophylactic cardiac medication initiated at least 1 year prior to LVD onset, and patients who received prophylactic cardiac medication were younger at their first encounter on average and were followed for a longer period. The most common medication class prescribed prophylactically was ACE inhibitors (ACEi), followed by angiotensin II receptor blockers (ARBs) and beta-blockers, with 86.7%, 7%, and 7% of patients receiving these medication classes, respectively. Among the patients treated prophylactically, 26.7% had multiple cardiac medication classes prescribed at some point.

Prophylactic treatment was associated with prolonged survival when adjusting for age at first visit and MD STARnet site. These patients had a 54% lower hazard of death (HR, 0.46; 95% CI, 0.22-0.93) compared with those who did not receive prophylactic cardiac medication. This finding was consistent even when adjusting for clinical covariates in supplemental analyses.

“Numerous studies support the benefit of prophylaxis on LV function but do not directly demonstrate survival benefit, in part because of the need to follow patients through their lifespan,” the authors wrote. “Our demonstration of prolonged survival associated with prophylactic cardiac medication is consistent with the few previous studies.”

The study had several limitations, including its reliance on surveillance data from medical records in MD STARnet sites. Medications were only reported annually, and the researchers presumed stop dates once medications were no longer listed. Another limitation was reliance on echocardiogram to determine LVD status, as more advanced imaging could potentially have shown abnormalities that echocardiogram did not show.

“The MD STARnet data show that treating individuals with DMD with first-line cardiac medications (ACEi or ARB) during the period when LV function is normal by echocardiogram is associated with delayed onset of LVD and prolonged survival in a large, population-based sample,” the authors concluded. “During the time period of this study, only one-quarter of individuals received this treatment, suggesting a focus on improving care.”

References

1. Conway KM, Thomas S, Neyaz T, et al. Prophylactic use of cardiac medications and survival in Duchenne muscular dystrophy. Muscle Nerve. Published online January 24, 2025. doi:10.1002/mus.28353

2. Muscular dystrophy research and tracking. CDC. Accessed January 24, 2025. https://www.cdc.gov/muscular-dystrophy/research/index.html