Skip to main content
Advertisement
  • Neurology.org
  • Journals
    • Neurology
    • Clinical Practice
    • Genetics
    • Neuroimmunology & Neuroinflammation
  • Specialty Sites
    • Equity, Diversity and Inclusion
    • Innovations in Care Delivery
    • Without Borders
  • Collections
    • Topics A-Z
    • Residents & Fellows
    • Infographics
    • Patient Pages
    • Null Hypothesis
    • Translations
  • Podcast
  • CME
  • About
    • About the Journals
    • Contact Us
    • Editorial Board
  • Authors
    • Submit a Manuscript
    • Author Center

Advanced Search

Main menu

  • Neurology.org
  • Journals
    • Neurology
    • Clinical Practice
    • Genetics
    • Neuroimmunology & Neuroinflammation
  • Specialty Sites
    • Equity, Diversity and Inclusion
    • Innovations in Care Delivery
    • Without Borders
  • Collections
    • Topics A-Z
    • Residents & Fellows
    • Infographics
    • Patient Pages
    • Null Hypothesis
    • Translations
  • Podcast
  • CME
  • About
    • About the Journals
    • Contact Us
    • Editorial Board
  • Authors
    • Submit a Manuscript
    • Author Center
  • Home
  • Latest Articles
  • Current Issue
  • Past Issues
  • Practice Current
  • Practice Buzz

User menu

  • Subscribe
  • My Alerts
  • Log in

Search

  • Advanced search
Neurology Clinical Practice
Home
A peer-reviewed clinical neurology journal for the practicing neurologist
  • Subscribe
  • My Alerts
  • Log in
Site Logo
  • Home
  • Latest Articles
  • Current Issue
  • Past Issues
  • Practice Current
  • Practice Buzz

Share

December 03, 2020Research

Preventing cardiomyopathy in DMD: A randomized, placebo-controlled drug-trial

John P. Bourke, Gillian Watson, View ORCID ProfileStefan Spinty, Andrew Bryant, View ORCID ProfileHelen Roper, Thomas Chadwick, Ruth Wood, View ORCID ProfileElaine McColl, Kate Bushby, View ORCID ProfileFrancesco Muntoni, Michela Guglieri; for the DMD Heart Study Group
First published December 3, 2020, DOI: https://doi.org/10.1212/CPJ.0000000000001023
John P. Bourke
1.Department of Cardiology, Freeman Hospital, NUTH NHS Hospitals Foundation Trust, Newcastle upon Tyne, UK. ()
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • For correspondence: john.bourke@nhs.net
Gillian Watson
2.Clinical Trials Unit, Newcastle University, Newcastle upon Tyne, UK. ()
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • For correspondence: gillian.watson@ncl.ac.uk
Stefan Spinty
3.Department of Paediatric Neurology, Alder Hey Children's NHS Foundation Trust, Liverpool, UK. ()
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • ORCID record for Stefan Spinty
  • For correspondence: stefan.spinty@alderhay.nhs.uk
Andrew Bryant
4.Population Health Sciences Institute, Newcastle University, Newcastle upon Tyne, UK. (; ; ).
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • For correspondence: andy.bryant@ncl.ac.uk thomas.chadwick@ncl.ac.uk elaine.mccoll@ncl.ac.uk
Helen Roper
5 Department of Paediatrics, Birmingham Heartlands Hospital, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK. ()
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • ORCID record for Helen Roper
  • For correspondence: helenroper1@googlemail.com
Thomas Chadwick
4.Population Health Sciences Institute, Newcastle University, Newcastle upon Tyne, UK. (; ; ).
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • For correspondence: andy.bryant@ncl.ac.uk thomas.chadwick@ncl.ac.uk elaine.mccoll@ncl.ac.uk
Ruth Wood
2.Clinical Trials Unit, Newcastle University, Newcastle upon Tyne, UK. ()
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • For correspondence: gillian.watson@ncl.ac.uk
Elaine McColl
4.Population Health Sciences Institute, Newcastle University, Newcastle upon Tyne, UK. (; ; ).
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • ORCID record for Elaine McColl
  • For correspondence: andy.bryant@ncl.ac.uk thomas.chadwick@ncl.ac.uk elaine.mccoll@ncl.ac.uk
Kate Bushby
6 John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle upon Tyne, NHS Hospitals Foundation Trust, UK. ; )
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • For correspondence: michela.guglieri@ncl.ac.uk kmds@me.com
Francesco Muntoni
7. NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, London, UK. ()
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • ORCID record for Francesco Muntoni
  • For correspondence: f.muntoni@ucl.ac.uk
Michela Guglieri
6 John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle upon Tyne, NHS Hospitals Foundation Trust, UK. ; )
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • For correspondence: michela.guglieri@ncl.ac.uk kmds@me.com
Full PDF
Citation
Preventing cardiomyopathy in DMD: A randomized, placebo-controlled drug-trial
John P. Bourke, Gillian Watson, Stefan Spinty, Andrew Bryant, Helen Roper, Thomas Chadwick, Ruth Wood, Elaine McColl, Kate Bushby, Francesco Muntoni, Michela Guglieri
Neurol Clin Pract Dec 2020, 10.1212/CPJ.0000000000001023; DOI: 10.1212/CPJ.0000000000001023

Citation Manager Formats

  • BibTeX
  • Bookends
  • EasyBib
  • EndNote (tagged)
  • EndNote 8 (xml)
  • Medlars
  • Mendeley
  • Papers
  • RefWorks Tagged
  • Ref Manager
  • RIS
  • Zotero
Permissions

Make Comment

See Comments

Downloads
28

Share

  • Article
  • Info & Disclosures
Loading

Abstract

Abstract

Objective: To determine whether a combination of two heart medications would be tolerated and could prevent / delay the onset of cardiomyopathy in boys with DMD compared to placebo.

Methods: This multi-centre, parallel group,1:1 patient randomised, placebo-controlled study of prophylactic perindopril and bisoprolol recruited boys with DMD aged 5-13 years, with normal ventricular function. Repeat assessments of LV-function, ECG and Adverse Event reporting were performed six-monthly. The primary outcome was change in ejection fraction between arms after 36 months. The study was approved by the NRES Committee East Midlands - Derby.

Results: Eighty-five boys were recruited (76% on steroid therapy) and randomised to combination heart drugs or matched placebo. Group change in LVEF% at 36 months from baseline was -2.2 + 6.0% and -2.9 + 6.1% in active and placebo arms (adjusted mean difference: -2.1, 95% CI -5.2 to 1.1). There was no difference between arms over repeated assessments (ANOVA) up to 36 months (trial arms p = 0.53); arm-over-time (p = 0.44). Four participants on placebo but none on active therapy were withdrawn due to deteriorations in LV-function. Secondary outcomes did not differ between arms either. Thirty-six Serious Adverse Events occurred none due to cardiac events or trial medication.

Conclusions: Combination therapy was well tolerated. Consistent with the previous prophylactic perindopril heart study, there was no evidence of group benefit after 36-months treatment.

Classification of evidence: This study provides Class 1 evidence that combination perindopril-bisoprolol therapy was well tolerated but did not change decline in LVEF significantly in boys with DMD.

  • Received June 28, 2020.
  • Accepted November 6, 2020.
  • © 2020 American Academy of Neurology

AAN Members

We have changed the login procedure to improve access between AAN.com and the Neurology journals. If you are experiencing issues, please log out of AAN.com and clear history and cookies. (For instructions by browser, please click the instruction pages below). After clearing, choose preferred Journal and select login for AAN Members. You will be redirected to a login page where you can log in with your AAN ID number and password. When you are returned to the Journal, your name should appear at the top right of the page.

Google Safari Microsoft Edge Firefox

Click here to login

AAN Non-Member Subscribers

Click here to login

Purchase access

For assistance, please contact:
AAN Members (800) 879-1960 or (612) 928-6000 (International)
Non-AAN Member subscribers (800) 638-3030 or (301) 223-2300 option 3, select 1 (international)

Sign Up
Information on how to subscribe to Neurology and Neurology: Clinical Practice can be found here 

Purchase
Individual access to articles is available through the Add to Cart option on the article page.  Access for 1 day (from the computer you are currently using) is US$ 39.00.  Pay-per-view content is for the use of the payee only, and content may not be further distributed by print or electronic means.  The payee may view, download, and/or print the article for his/her personal, scholarly, research, and educational use.  Distributing copies (electronic or otherwise) of the article is not allowed.

The Nerve!: Rapid online correspondence

No comments have been published for this article.
Comment

NOTE: All contributors' disclosures must be entered and current in our database before comments can be posted. Enter and update disclosures at http://submit.cp.neurology.org. Exception: replies to comments concerning an article you originally authored do not require updated disclosures.

  • Stay timely. Submit only on articles published within the last 8 weeks.
  • Do not be redundant. Read any comments already posted on the article prior to submission.
  • 200 words maximum.
  • 5 references maximum. Reference 1 must be the article on which you are commenting.
  • 5 authors maximum. Exception: replies can include all original authors of the article.
  • Submitted comments are subject to editing and editor review prior to posting.

More guidelines and information on The Nerve!

Compose Comment

More information about text formats

Plain text

  • No HTML tags allowed.
  • Web page addresses and e-mail addresses turn into links automatically.
  • Lines and paragraphs break automatically.
Author Information
NOTE: The first author must also be the corresponding author of the comment.
First or given name, e.g. 'Peter'.
Your last, or family, name, e.g. 'MacMoody'.
Your email address, e.g. higgs-boson@gmail.com
Your role and/or occupation, e.g. 'Orthopedic Surgeon'.
Your organization or institution (if applicable), e.g. 'Royal Free Hospital'.
Publishing Agreement
NOTE: All authors, besides the first/corresponding author, must complete a separate The Nerve! Submission Form and provide via email to the editorial office before comments can be posted.
CAPTCHA
This question is for testing whether or not you are a human visitor and to prevent automated spam submissions.

Vertical Tabs

You May Also be Interested in

Back to top
  • Article
  • Info & Disclosures
Advertisement

Related Articles

  • No related articles found.

Topics Discussed

  • All Pediatric
  • Clinical trials Randomized controlled (CONSORT agreement)
  • Muscle disease

Alert Me

  • Alert me when eletters are published
Neurology: Clinical Practice: 11 (2)

Articles

  • Ahead of Print
  • Current Issue
  • Past Issues
  • Popular Articles

About

  • About the Journals
  • Ethics Policies
  • Editors & Editorial Board
  • Contact Us
  • Advertise

Submit

  • Author Center
  • Submit a Manuscript
  • Information for Reviewers
  • AAN Guidelines
  • Permissions

Subscribers

  • Subscribe
  • Activate a Subscription
  • Sign up for eAlerts
  • RSS Feed
Site Logo
  • Visit neurology Template on Facebook
  • Follow neurology Template on Twitter
  • Visit Neurology on YouTube
  • Neurology
  • Neurology: Clinical Practice
  • Neurology: Genetics
  • Neurology: Neuroimmunology & Neuroinflammation
  • AAN.com
  • AANnews
  • Continuum
  • Brain & Life
  • Neurology Today

Wolters Kluwer Logo

Neurology: Clinical Practice |  Print ISSN: 2163-0402
Online ISSN: 2163-0933

© 2021 American Academy of Neurology

  • Privacy Policy
  • Feedback
  • Advertise