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Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy: A Randomized Clinical Trial.

Publication ,  Journal Article
Silva, MC; Magalhães, TA; Meira, ZMA; Rassi, CHRE; Andrade, ACDS; Gutierrez, PS; Azevedo, CF; Gurgel-Giannetti, J; Vainzof, M; Zatz, M ...
Published in: JAMA cardiology
February 2017

In Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), interventions reducing the progression of myocardial disease could affect survival.To assess the effect of early angiotensin-converting enzyme (ACE) inhibitor therapy in patients with normal left ventricular function on the progression of myocardial fibrosis (MF) identified on cardiovascular magnetic resonance (CMR).A randomized clinical trial conducted in 2 centers included 76 male patients with DMD or BMD undergoing 2 CMR studies with a 2-year interval for ventricular function and MF assessment. In a non-intent-to-treat trial, 42 patients with MF and normal left ventricular ejection fraction (LVEF) were randomized (1:1) to receive or not receive ACE inhibitor therapy. The study was conducted from June 26, 2009, to June 30, 2012. Data analysis was performed from June 30, 2013, to October 3, 2016.Randomization (1:1) to receive or not receive ACE inhibitor therapy.Primary outcome was MF progression from baseline to the 2-year CMR study.Of the 76 male patients included in the study, 70 had DMD (92%) and 6 had BMD (8%); mean (SD) age at baseline was 13.1 (4.4) years. Myocardial fibrosis was present in 55 patients (72%) and LV systolic dysfunction was identified in 13 patients (24%). Myocardial fibrosis at baseline was an independent indicator of lower LVEF at follow-up (coefficient [SE], -0.16 [0.07]; P = .03). Among patients with MF and preserved LVEF (42 [55%]), those randomized (21 patients in each arm) to receive ACE inhibitors demonstrated slower MF progression compared with the untreated group (mean [SD] increase of 3.1% [7.4%] vs 10.0% [6.2%] as a percentage of LV mass; P = .001). In multivariate analysis, ACE inhibitor therapy was an independent indicator of decreased MF progression (coefficient [SE], -4.51 [2.11]; P = .04). Patients with MF noted on CMR had a higher probability of cardiovascular events (event rate, 10 of 55 [18.2%] vs 0 of 21 [0%]; log-rank P = .04).In this 2-year, follow-up, randomized clinical trial of patients with Duchenne or Becker muscular dystrophy whose LVEF was preserved and MF was present as determined on CMR, ACE inhibitor therapy was associated with significantly slower progression of MF. The presence of MF was associated with worse patient prognosis.clinicaltrials.org Identifier: NCT02432885.

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Published In

JAMA cardiology

DOI

EISSN

2380-6591

ISSN

2380-6583

Publication Date

February 2017

Volume

2

Issue

2

Start / End Page

190 / 199

Related Subject Headings

  • Stroke Volume
  • Registries
  • Prospective Studies
  • Prognosis
  • Myocardium
  • Muscular Dystrophy, Duchenne
  • Male
  • Magnetic Resonance Imaging, Cine
  • Humans
  • Follow-Up Studies
 

Citation

APA
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Silva, M. C., Magalhães, T. A., Meira, Z. M. A., Rassi, C. H. R. E., Andrade, A. C. D. S., Gutierrez, P. S., … Rochitte, C. E. (2017). Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy: A Randomized Clinical Trial. JAMA Cardiology, 2(2), 190–199. https://doi.org/10.1001/jamacardio.2016.4801
Silva, Marly Conceição, Tiago Augusto Magalhães, Zilda Maria Alves Meira, Carlos Henrique Reis Esselin Rassi, Amanda Cristina de Souza Andrade, Paulo Sampaio Gutierrez, Clerio Francisco Azevedo, et al. “Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy: A Randomized Clinical Trial.JAMA Cardiology 2, no. 2 (February 2017): 190–99. https://doi.org/10.1001/jamacardio.2016.4801.
Silva MC, Magalhães TA, Meira ZMA, Rassi CHRE, Andrade ACDS, Gutierrez PS, et al. Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy: A Randomized Clinical Trial. JAMA cardiology. 2017 Feb;2(2):190–9.
Silva, Marly Conceição, et al. “Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy: A Randomized Clinical Trial.JAMA Cardiology, vol. 2, no. 2, Feb. 2017, pp. 190–99. Epmc, doi:10.1001/jamacardio.2016.4801.
Silva MC, Magalhães TA, Meira ZMA, Rassi CHRE, Andrade ACDS, Gutierrez PS, Azevedo CF, Gurgel-Giannetti J, Vainzof M, Zatz M, Kalil-Filho R, Rochitte CE. Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy: A Randomized Clinical Trial. JAMA cardiology. 2017 Feb;2(2):190–199.

Published In

JAMA cardiology

DOI

EISSN

2380-6591

ISSN

2380-6583

Publication Date

February 2017

Volume

2

Issue

2

Start / End Page

190 / 199

Related Subject Headings

  • Stroke Volume
  • Registries
  • Prospective Studies
  • Prognosis
  • Myocardium
  • Muscular Dystrophy, Duchenne
  • Male
  • Magnetic Resonance Imaging, Cine
  • Humans
  • Follow-Up Studies