Use of advanced heart failure therapies in Duchenne muscular dystrophy

Carol A. Wittlieb-Weber*, Chet R. Villa, Jennifer Conway, Matthew J. Bock, Katheryn E Gambetta, Jonathan N. Johnson, Ashwin K. Lal, Kurt R. Schumacher, Sabrina P. Law, Shriprasad R. Deshpande, Shawn C. West, Joshua M. Friedland-Little, Irene D. Lytrivi, Michael A. McCulloch, Ryan J. Butts, David R. Weber, Kenneth R. Knecht

*Corresponding author for this work

Research output: Contribution to journalArticle

1 Citation (Scopus)

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.

Original languageEnglish (US)
Pages (from-to)11-14
Number of pages4
JournalProgress in Pediatric cardiology
Volume53
DOIs
StatePublished - Jun 1 2019

Fingerprint

Heart-Assist Devices
Duchenne Muscular Dystrophy
Implantable Defibrillators
Heart Failure
Therapeutics
Respiratory Therapy
Heart Transplantation
Ventricular Tachycardia
Glucocorticoids
Shock
Cohort Studies
Retrospective Studies
Population

Keywords

  • Duchenne muscular dystrophy
  • Dystrophic cardiomyopathy
  • Heart failure

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Cardiology and Cardiovascular Medicine

Cite this

Wittlieb-Weber, C. A., Villa, C. R., Conway, J., Bock, M. J., Gambetta, K. E., Johnson, J. N., ... Knecht, K. R. (2019). Use of advanced heart failure therapies in Duchenne muscular dystrophy. Progress in Pediatric cardiology, 53, 11-14. https://doi.org/10.1016/j.ppedcard.2019.01.001
Wittlieb-Weber, Carol A. ; Villa, Chet R. ; Conway, Jennifer ; Bock, Matthew J. ; Gambetta, Katheryn E ; Johnson, Jonathan N. ; Lal, Ashwin K. ; Schumacher, Kurt R. ; Law, Sabrina P. ; Deshpande, Shriprasad R. ; West, Shawn C. ; Friedland-Little, Joshua M. ; Lytrivi, Irene D. ; McCulloch, Michael A. ; Butts, Ryan J. ; Weber, David R. ; Knecht, Kenneth R. / Use of advanced heart failure therapies in Duchenne muscular dystrophy. In: Progress in Pediatric cardiology. 2019 ; Vol. 53. pp. 11-14.
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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.",
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Wittlieb-Weber, CA, Villa, CR, Conway, J, Bock, MJ, Gambetta, KE, Johnson, JN, Lal, AK, Schumacher, KR, Law, SP, Deshpande, SR, West, SC, Friedland-Little, JM, Lytrivi, ID, McCulloch, MA, Butts, RJ, Weber, DR & Knecht, KR 2019, 'Use of advanced heart failure therapies in Duchenne muscular dystrophy', Progress in Pediatric cardiology, vol. 53, pp. 11-14. https://doi.org/10.1016/j.ppedcard.2019.01.001

Use of advanced heart failure therapies in Duchenne muscular dystrophy. / Wittlieb-Weber, Carol A.; Villa, Chet R.; Conway, Jennifer; Bock, Matthew J.; Gambetta, Katheryn E; Johnson, Jonathan N.; Lal, Ashwin K.; Schumacher, Kurt R.; Law, Sabrina P.; Deshpande, Shriprasad R.; West, Shawn C.; Friedland-Little, Joshua M.; Lytrivi, Irene D.; McCulloch, Michael A.; Butts, Ryan J.; Weber, David R.; Knecht, Kenneth R.

In: Progress in Pediatric cardiology, Vol. 53, 01.06.2019, p. 11-14.

Research output: Contribution to journalArticle

TY - JOUR

T1 - Use of advanced heart failure therapies in Duchenne muscular dystrophy

AU - Wittlieb-Weber, Carol A.

AU - Villa, Chet R.

AU - Conway, Jennifer

AU - Bock, Matthew J.

AU - Gambetta, Katheryn E

AU - Johnson, Jonathan N.

AU - Lal, Ashwin K.

AU - Schumacher, Kurt R.

AU - Law, Sabrina P.

AU - Deshpande, Shriprasad R.

AU - West, Shawn C.

AU - Friedland-Little, Joshua M.

AU - Lytrivi, Irene D.

AU - McCulloch, Michael A.

AU - Butts, Ryan J.

AU - Weber, David R.

AU - Knecht, Kenneth R.

PY - 2019/6/1

Y1 - 2019/6/1

N2 - 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.

AB - 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.

KW - Duchenne muscular dystrophy

KW - Dystrophic cardiomyopathy

KW - Heart failure

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