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.
N1 - Funding Information:
Database creation and management was funded by the Division of Pediatric Cardiology at Golisano Children's Hospital, University of Rochester Medical Center , Rochester, New York. David R. Weber received salary support from NIH grant K23DK114477 .
Funding Information:
Database creation and management was funded by the Division of Pediatric Cardiology at Golisano Children's Hospital, University of Rochester Medical Center, Rochester, New York. David R. Weber received salary support from NIH grant K23DK114477.
Publisher Copyright:
© 2019 Elsevier B.V.
PY - 2019/6
Y1 - 2019/6
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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U2 - 10.1016/j.ppedcard.2019.01.001
DO - 10.1016/j.ppedcard.2019.01.001
M3 - Article
C2 - 31360053
AN - SCOPUS:85059745953
SN - 1058-9813
VL - 53
SP - 11
EP - 14
JO - Progress in Pediatric Cardiology
JF - Progress in Pediatric Cardiology
ER -