Purpose
Cardiac disease results in significant morbidity and mortality in patients with muscular dystrophy (MD). Single centers have reported their ventricular assist device (VAD) experience in specific MDs and in limited numbers. This study sought to describe the outcomes associated with VAD therapy in an unselected population across multiple centers.
Methods
We examined outcomes of patients with MD and dilated cardiomyopathy implanted with a VAD at Advanced Cardiac Therapies Improving Outcomes Network (ACTION) centers from 9/2012 to 9/2020.
Results
A total of 19 VADs were implanted in 18 patients across 12 sites. The majority of patients had dystrophinopathy (66%) and the median age at implant was 17.2 years (range 11.7-29.5). Eleven patients were non-ambulatory (61%) and 6 (33%) were on respiratory support pre-VAD. Five (28%) patients were implanted as a bridge to transplant, 4 of whom survived to transplant. Of 13 patients implanted as bridge to decision or destination therapy, 77% were alive at 1 year and 69% at 2 years. The overall frequencies of positive outcome (transplanted or alive on device) at 1 year and 2 years were 84% and 78%, respectively. Two patients suffered a stroke, 2 developed sepsis, 1 required tracheostomy, and 1 experienced severe right heart failure requiring right-sided VAD.
Conclusions
This study demonstrates the potential utility of VAD therapies in patients with muscular dystrophy. Further research is needed to further improve outcomes and better determine which patients may benefit most from VAD therapy in terms of survival and quality of life.
This ACTION study was published in September 2022 in The Journal of Heart and Lung Transplantation. Congratulations to the researchers:
- Deipanjan Nandi, MD, MSc
- Scott R. Auerbach, MD
- Neha Bansal, MD
- Holger Buchholz, MD
- Jennifer Conway, MD
- Paul Esteso, MD, PhD
- Beth D. Kaufman, MD
- Ashwin K. Lal, MD
- Sabrina P. Law, MD
- Angela Lorts, MD, MBA,
- Lindsay J. May, MD
- Mary Mehegan, BSN, RN, CCRN
- Deepa Mokshagundam, MD
- David L.S. Morales, MD
- Matthew J. O’Connor, MD
- David N. Rosenthal, MD
- Muhammad F. Shezad, MBBS, MHSA
- Kathleen E. Simpson, MD
- David L. Sutcliffe, MD
- Christina Vanderpluym, MD
- Carol A. Wittlieb-Weber, MD
- Farhan Zafar, MD
- Linda Cripe, MD
- Chet R. Villa, MD
CITATION
Nandi D, Auerbach SR, Bansal N, Buchholz H, Conway J, Esteso P, Kaufman BD, Lal AK, Law SP, Lorts A, May LJ, Mehegan M, Mokshagundam D, Morales DLS, O’Connor MJ, Rosenthal DN, Shezad MF, Simpson KE, Sutcliffe DL, Vanderpluym C, Wittlieb-Weber CA, Zafar F, Cripe L and Villa CR. Initial multicenter experience with ventricular assist devices in children and young adults with muscular dystrophy: An ACTION registry analysis. J Heart Lung Transplant. 2022.
