Related Experiment Video
Updated: Dec 25, 2025
Purification and Transplantation of Myogenic Progenitor Cell Derived Exosomes to Improve Cardiac Function in Duchenne Muscular Dystrophic Mice
Published on: April 10, 2019
Heart Transplantation in Muscular Dystrophy Patients: Is it a Viable Option?
Dennis Wells1, Raheel Rizwan2, John L Jefferies3,2
1From the Department of Cardiothoracic Surgery (D.W.), University of Cincinnati College of Medicine, OH.
Insights
Heart transplantation (HTx) outcomes for muscular dystrophy (MD) patients show similar survival rates compared to other cardiomyopathy patients. This suggests HTx is an effective treatment for select MD patients with end-stage heart failure.
Area of Science:
- Cardiology
- Transplantation Medicine
- Genetics
Background:
- Cardiomyopathy is a frequent and serious complication in muscular dystrophy (MD) patients, often leading to advanced heart failure and early mortality.
- Despite the severity, there's a notable reluctance to consider heart transplantation (HTx) for these individuals.
- This study investigates the outcomes of HTx in patients diagnosed with MD within the United States.
Purpose of the Study:
- To evaluate the outcomes of heart transplantation (HTx) in patients with muscular dystrophy (MD).
- To compare the post-transplant survival rates of MD patients with those of other cardiomyopathy patients.
- To assess the efficacy of HTx as a treatment for end-stage heart failure in select MD patients.
Main Methods:
- Data from the United Network for Organ Sharing database (October 1987–March 2016) were analyzed.
- A cohort of 81 MD patients undergoing HTx was compared with a control group of 41,317 other cardiomyopathy patients.
- Propensity score matching was employed to create a comparable cohort of 162 patients, controlling for age, gender, transplant year, renal function, and inotropic support.
Main Results:
- Pre-transplant characteristics were similar between MD and matched cardiomyopathy cohorts, with exceptions in ventricular assist device use, ventilator support, and donor race mismatch.
- Median waitlist time did not differ significantly between the groups.
- Post-transplant survival for the MD cohort was comparable to the matched cardiomyopathy cohort (P=0.18) and superior to the unmatched cardiomyopathy cohort (P=0.004).
Conclusions:
- Patients with muscular dystrophy who undergo heart transplantation experience long-term survival rates similar to recipients with other forms of cardiomyopathy.
- Heart transplantation is a viable and effective treatment option for carefully selected muscular dystrophy patients suffering from end-stage heart failure.
- No significant difference in post-transplant survival was observed between Becker MD and non-Becker MD subtypes.
Background:
Cardiomyopathy is a common complication among muscular dystrophy (MD) patients and often results in advanced heart failure and premature death. In spite of this, there is hesitancy to consider heart transplantation (HTx). This study describes the HTx outcomes in patients with MD in the United States.
Methods And Results:
All HTx in the United Network for Organ Sharing database from October 1, 1987, to March 31, 2016, were identified. Two patient groups were created: MD cohort (n=81), and a cohort of all other cardiomyopathies, called cardiomyopathy-unmatched (n=41 317). Propensity score matching (ratio 1:2) was performed on transplant age, gender transplant year, renal function, and inotropic support at transplant to form a cardiomyopathy-matched cohort (n=162). Patient characteristics and posttransplant outcomes were compared. In the 81 patients with MD, Becker was the most common type (42%-52%). All the analyzed preoperative characteristics did not statistically differ between the MD and cardiomyopathy-matched cohorts except ventricular assist device use (16% versus 30%; P=0.017), ventilator support (0% versus 6%; P=0.031), and donor race mismatch (30% versus 55%; P<0.001). Median time on waitlist was not statistically different between the 2 groups (52 versus 59 days; P=0.12). Posttransplant survival of MD cohort was not statistically different compared with cardiomyopathy-matched cohort (P=0.18; hazard ratio [95% CI], 0.71 [0.42-1.18]) and was better than the cardiomyopathy-unmatched cohort (P=0.004; hazard ratio [95% CI], 0.53 [0.34-0.82]). Among the types of MD, no statistical difference was observed in posttransplant survival of Becker MD versus non-Becker MD (P=0.12; hazard ratio [95% CI], 2.17 [0.79-6.01]).
Conclusions:
Patients with MD undergoing HTx had similar long-term posttransplant survival compared with matched cardiomyopathy-related HTx recipients. HTx appears to be an effective treatment for a select group of muscular dystrophy patients with end-stage heart failure.
Related Concept Videos
01:21Satellite Stem Cells and Muscular Dystrophy
01:30Cardiomyopathy II: Dilated Cardiomyopathy
01:29Cardiomyopathy V: Interprofessional Care