Carol A Wittlieb-Weber1, Chet R Villa2, Jennifer Conway3, Matthew J Bock4, Katheryn E Gambetta5, Jonathan N Johnson6, Ashwin K Lal7, Kurt R Schumacher8, Sabrina P Law9, Shriprasad R Deshpande10, Shawn C West11, Joshua M Friedland-Little12, Irene D Lytrivi9, Michael A McCulloch13, Ryan J Butts14, David R Weber1, Kenneth R Knecht15. 1. Golisano Children's Hospital, University of Rochester Medical Center, Rochester, NY, United States of America. 2. The Heart Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, United States of America. 3. Stollery Children's Hospital, University of Alberta, Edmonton, Alberta, Canada. 4. Loma Linda University Children's Hospital, Loma Linda, CA, United States of America. 5. Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, IL, United States of America. 6. Mayo Clinic Children's Center, Rochester, MN, United States of America. 7. Primary Children's Hospital, University of Utah, Salt Lake City, UT, United States of America. 8. C.S. Mott Children's Hospital, University of Michigan, Ann Arbor, MI, United States of America. 9. Morgan Stanley Children's Hospital of New York Presbyterian, New York, NY, United States of America. 10. Children's National, Washington, DC, United States of America. 11. Children's Hospital of Pittsburgh of UPMC, Pittsburgh, PA, United States of America. 12. Seattle Children's Hospital, Seattle, WA, United States of America. 13. University of Virginia Children's Hospital, Charlottesville, VA, United States of America. 14. Children's Medical Center of Dallas, UT Southwestern Medical Center, Dallas, TX, United States of America. 15. Arkansas Children's Hospital, University of Arkansas for Medical Sciences, Little Rock, AR, United States of America.
Abstract
BACKGROUND: As survival and neuromuscular function in Duchenne Muscular Dystrophy (DMD) improve with glucocorticoid therapy and respiratory advances, the proportion of cardiac deaths is increasing. Little is known about the use and outcomes of advanced heart failure (HF) therapies in this population. METHODS: A retrospective cohort study of 436 males with DMD was performed, from January 1, 2005-January 1, 2018, with the primary outcome being use of advanced HF therapies including: implantable cardioverter defibrillator (ICD), left ventricular assist device (LVAD), and heart transplantation (HTX). RESULTS: Nine subjects had an ICD placed, 2 of whom (22.2%) had appropriate shocks for ventricular tachycardia; 1 and 968 days after implant, and all of whom were alive at last follow-up; median 18 (IQR: 12.5-25.5) months from implant. Four subjects had a LVAD implanted with post-LVAD survival of 75% at 1 year; 2 remaining on support and 1 undergoing HTX. One subject was bridged to HTX with ICD and LVAD and was alive at last follow-up, 53 months after HTX. CONCLUSION: Advanced HF therapies may be used effectively in select subjects with DMD. Further studies are needed to better understand risk stratification for ICD use and optimal candidacy for LVAD implantation and HTX, with hopes of improving cardiac outcomes.
BACKGROUND: As survival and neuromuscular function in Duchenne Muscular Dystrophy (DMD) improve with glucocorticoid therapy and respiratory advances, the proportion of cardiac deaths is increasing. Little is known about the use and outcomes of advanced heart failure (HF) therapies in this population. METHODS: A retrospective cohort study of 436 males with DMD was performed, from January 1, 2005-January 1, 2018, with the primary outcome being use of advanced HF therapies including: implantable cardioverter defibrillator (ICD), left ventricular assist device (LVAD), and heart transplantation (HTX). RESULTS: Nine subjects had an ICD placed, 2 of whom (22.2%) had appropriate shocks for ventricular tachycardia; 1 and 968 days after implant, and all of whom were alive at last follow-up; median 18 (IQR: 12.5-25.5) months from implant. Four subjects had a LVAD implanted with post-LVAD survival of 75% at 1 year; 2 remaining on support and 1 undergoing HTX. One subject was bridged to HTX with ICD and LVAD and was alive at last follow-up, 53 months after HTX. CONCLUSION: Advanced HF therapies may be used effectively in select subjects with DMD. Further studies are needed to better understand risk stratification for ICD use and optimal candidacy for LVAD implantation and HTX, with hopes of improving cardiac outcomes.
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