Skip to main content
Advertisement
  • Neurology.org
  • Journals
    • Neurology
    • Clinical Practice
    • Education
    • Genetics
    • Neuroimmunology & Neuroinflammation
  • Online Sections
    • Neurology Video Journal Club
    • Diversity, Equity, & Inclusion (DEI)
    • Neurology: Clinical Practice Accelerator
    • Practice Buzz
    • Practice Current
    • Residents & Fellows
    • Without Borders
  • Collections
    • COVID-19
    • Disputes & Debates
    • Health Disparities
    • Infographics
    • Null Hypothesis
    • Patient Pages
    • Topics A-Z
    • Translations
    • UDDA Revision Series
  • Podcast
  • CME
  • About
    • About the Journals
    • Contact Us
    • Editorial Board
  • Authors
    • Submit Manuscript
    • Author Center

Advanced Search

Main menu

  • Neurology.org
  • Journals
    • Neurology
    • Clinical Practice
    • Education
    • Genetics
    • Neuroimmunology & Neuroinflammation
  • Online Sections
    • Neurology Video Journal Club
    • Diversity, Equity, & Inclusion (DEI)
    • Neurology: Clinical Practice Accelerator
    • Practice Buzz
    • Practice Current
    • Residents & Fellows
    • Without Borders
  • Collections
    • COVID-19
    • Disputes & Debates
    • Health Disparities
    • Infographics
    • Null Hypothesis
    • Patient Pages
    • Topics A-Z
    • Translations
    • UDDA Revision Series
  • Podcast
  • CME
  • About
    • About the Journals
    • Contact Us
    • Editorial Board
  • Authors
    • Submit Manuscript
    • Author Center
  • Home
  • Articles
  • 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
  • Articles
  • Issues
  • Practice Current
  • Practice Buzz

Share

October 2021; 11 (5) Research

Preventing Cardiomyopathy in DMD

A Randomized Placebo-Controlled Drug Trial

John P. Bourke, View ORCID ProfileGillian 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
Department of Cardiology (JPB), Freeman Hospital, NUTH NHS Hospitals Foundation Trust; Clinical Trials Unit (MC, RW), Newcastle University, Newcastle upon Tyne; Department of Paediatric Neurology (SS), Alder Hey Children's NHS Foundation Trust, Liverpool; Population Health Sciences Institute (AB, TC, EM), Newcastle University, Newcastle upon Tyne; Department of Paediatrics (HR), Birmingham Heartlands Hospital, University Hospitals Birmingham NHS Foundation Trust; John Walton Muscular Dystrophy Research Centre (KB, MG), Newcastle University and Newcastle upon Tyne, NHS Hospitals Foundation Trust, Newcastle upon Tyne; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, UK.
MD
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Gillian Watson
Department of Cardiology (JPB), Freeman Hospital, NUTH NHS Hospitals Foundation Trust; Clinical Trials Unit (MC, RW), Newcastle University, Newcastle upon Tyne; Department of Paediatric Neurology (SS), Alder Hey Children's NHS Foundation Trust, Liverpool; Population Health Sciences Institute (AB, TC, EM), Newcastle University, Newcastle upon Tyne; Department of Paediatrics (HR), Birmingham Heartlands Hospital, University Hospitals Birmingham NHS Foundation Trust; John Walton Muscular Dystrophy Research Centre (KB, MG), Newcastle University and Newcastle upon Tyne, NHS Hospitals Foundation Trust, Newcastle upon Tyne; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, UK.
MClinRes
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • ORCID record for Gillian Watson
Stefan Spinty
Department of Cardiology (JPB), Freeman Hospital, NUTH NHS Hospitals Foundation Trust; Clinical Trials Unit (MC, RW), Newcastle University, Newcastle upon Tyne; Department of Paediatric Neurology (SS), Alder Hey Children's NHS Foundation Trust, Liverpool; Population Health Sciences Institute (AB, TC, EM), Newcastle University, Newcastle upon Tyne; Department of Paediatrics (HR), Birmingham Heartlands Hospital, University Hospitals Birmingham NHS Foundation Trust; John Walton Muscular Dystrophy Research Centre (KB, MG), Newcastle University and Newcastle upon Tyne, NHS Hospitals Foundation Trust, Newcastle upon Tyne; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, UK.
MMedSc
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • ORCID record for Stefan Spinty
Andrew Bryant
Department of Cardiology (JPB), Freeman Hospital, NUTH NHS Hospitals Foundation Trust; Clinical Trials Unit (MC, RW), Newcastle University, Newcastle upon Tyne; Department of Paediatric Neurology (SS), Alder Hey Children's NHS Foundation Trust, Liverpool; Population Health Sciences Institute (AB, TC, EM), Newcastle University, Newcastle upon Tyne; Department of Paediatrics (HR), Birmingham Heartlands Hospital, University Hospitals Birmingham NHS Foundation Trust; John Walton Muscular Dystrophy Research Centre (KB, MG), Newcastle University and Newcastle upon Tyne, NHS Hospitals Foundation Trust, Newcastle upon Tyne; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, UK.
MSc
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Helen Roper
Department of Cardiology (JPB), Freeman Hospital, NUTH NHS Hospitals Foundation Trust; Clinical Trials Unit (MC, RW), Newcastle University, Newcastle upon Tyne; Department of Paediatric Neurology (SS), Alder Hey Children's NHS Foundation Trust, Liverpool; Population Health Sciences Institute (AB, TC, EM), Newcastle University, Newcastle upon Tyne; Department of Paediatrics (HR), Birmingham Heartlands Hospital, University Hospitals Birmingham NHS Foundation Trust; John Walton Muscular Dystrophy Research Centre (KB, MG), Newcastle University and Newcastle upon Tyne, NHS Hospitals Foundation Trust, Newcastle upon Tyne; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, UK.
MD
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • ORCID record for Helen Roper
Thomas Chadwick
Department of Cardiology (JPB), Freeman Hospital, NUTH NHS Hospitals Foundation Trust; Clinical Trials Unit (MC, RW), Newcastle University, Newcastle upon Tyne; Department of Paediatric Neurology (SS), Alder Hey Children's NHS Foundation Trust, Liverpool; Population Health Sciences Institute (AB, TC, EM), Newcastle University, Newcastle upon Tyne; Department of Paediatrics (HR), Birmingham Heartlands Hospital, University Hospitals Birmingham NHS Foundation Trust; John Walton Muscular Dystrophy Research Centre (KB, MG), Newcastle University and Newcastle upon Tyne, NHS Hospitals Foundation Trust, Newcastle upon Tyne; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, UK.
PhD
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Ruth Wood
Department of Cardiology (JPB), Freeman Hospital, NUTH NHS Hospitals Foundation Trust; Clinical Trials Unit (MC, RW), Newcastle University, Newcastle upon Tyne; Department of Paediatric Neurology (SS), Alder Hey Children's NHS Foundation Trust, Liverpool; Population Health Sciences Institute (AB, TC, EM), Newcastle University, Newcastle upon Tyne; Department of Paediatrics (HR), Birmingham Heartlands Hospital, University Hospitals Birmingham NHS Foundation Trust; John Walton Muscular Dystrophy Research Centre (KB, MG), Newcastle University and Newcastle upon Tyne, NHS Hospitals Foundation Trust, Newcastle upon Tyne; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, UK.
MSc
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Elaine McColl
Department of Cardiology (JPB), Freeman Hospital, NUTH NHS Hospitals Foundation Trust; Clinical Trials Unit (MC, RW), Newcastle University, Newcastle upon Tyne; Department of Paediatric Neurology (SS), Alder Hey Children's NHS Foundation Trust, Liverpool; Population Health Sciences Institute (AB, TC, EM), Newcastle University, Newcastle upon Tyne; Department of Paediatrics (HR), Birmingham Heartlands Hospital, University Hospitals Birmingham NHS Foundation Trust; John Walton Muscular Dystrophy Research Centre (KB, MG), Newcastle University and Newcastle upon Tyne, NHS Hospitals Foundation Trust, Newcastle upon Tyne; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, UK.
PhD
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • ORCID record for Elaine McColl
Kate Bushby
Department of Cardiology (JPB), Freeman Hospital, NUTH NHS Hospitals Foundation Trust; Clinical Trials Unit (MC, RW), Newcastle University, Newcastle upon Tyne; Department of Paediatric Neurology (SS), Alder Hey Children's NHS Foundation Trust, Liverpool; Population Health Sciences Institute (AB, TC, EM), Newcastle University, Newcastle upon Tyne; Department of Paediatrics (HR), Birmingham Heartlands Hospital, University Hospitals Birmingham NHS Foundation Trust; John Walton Muscular Dystrophy Research Centre (KB, MG), Newcastle University and Newcastle upon Tyne, NHS Hospitals Foundation Trust, Newcastle upon Tyne; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, UK.
PhD
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Francesco Muntoni
Department of Cardiology (JPB), Freeman Hospital, NUTH NHS Hospitals Foundation Trust; Clinical Trials Unit (MC, RW), Newcastle University, Newcastle upon Tyne; Department of Paediatric Neurology (SS), Alder Hey Children's NHS Foundation Trust, Liverpool; Population Health Sciences Institute (AB, TC, EM), Newcastle University, Newcastle upon Tyne; Department of Paediatrics (HR), Birmingham Heartlands Hospital, University Hospitals Birmingham NHS Foundation Trust; John Walton Muscular Dystrophy Research Centre (KB, MG), Newcastle University and Newcastle upon Tyne, NHS Hospitals Foundation Trust, Newcastle upon Tyne; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, UK.
MD
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • ORCID record for Francesco Muntoni
Michela Guglieri
Department of Cardiology (JPB), Freeman Hospital, NUTH NHS Hospitals Foundation Trust; Clinical Trials Unit (MC, RW), Newcastle University, Newcastle upon Tyne; Department of Paediatric Neurology (SS), Alder Hey Children's NHS Foundation Trust, Liverpool; Population Health Sciences Institute (AB, TC, EM), Newcastle University, Newcastle upon Tyne; Department of Paediatrics (HR), Birmingham Heartlands Hospital, University Hospitals Birmingham NHS Foundation Trust; John Walton Muscular Dystrophy Research Centre (KB, MG), Newcastle University and Newcastle upon Tyne, NHS Hospitals Foundation Trust, Newcastle upon Tyne; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, UK.
MD
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
Department of Cardiology (JPB), Freeman Hospital, NUTH NHS Hospitals Foundation Trust; Clinical Trials Unit (MC, RW), Newcastle University, Newcastle upon Tyne; Department of Paediatric Neurology (SS), Alder Hey Children's NHS Foundation Trust, Liverpool; Population Health Sciences Institute (AB, TC, EM), Newcastle University, Newcastle upon Tyne; Department of Paediatrics (HR), Birmingham Heartlands Hospital, University Hospitals Birmingham NHS Foundation Trust; John Walton Muscular Dystrophy Research Centre (KB, MG), Newcastle University and Newcastle upon Tyne, NHS Hospitals Foundation Trust, Newcastle upon Tyne; and NIHR Great Ormond Street Hospital Biomedical Research Centre (FM), Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, UK.
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, for the DMD Heart Study Group
Neurol Clin Pract Oct 2021, 11 (5) e661-e668; 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
120

Share

  • Article
  • Figures & Data
  • Info & Disclosures
Loading

This article requires a subscription to view the full text. If you have a subscription you may use the login form below to view the article. Access to this article can also be purchased.

Abstract

Objective To determine whether a combination of 2 heart medications would be tolerated and could prevent/delay the onset of cardiomyopathy in boys with Duchenne muscular dystrophy (DMD) compared with placebo.

Methods This multicenter, parallel group, 1:1 patient randomized, placebo-controlled study of prophylactic perindopril and bisoprolol recruited boys with DMD aged 5–13 years, with normal ventricular function. Repeat assessments of left ventricular (LV) function, electrocardiogram, and adverse event reporting were performed 6 monthly. The primary outcome was change in ejection fraction between arms after 36 months. The study was approved by the National Research Ethics Service Committee East Midlands—Derby.

Results Eighty-five boys were recruited (76% on steroid therapy) and randomized to combination heart drugs or matched placebo. Group change in left ventricular ejection fraction (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 treatment arms over repeated assessments (analysis of variance) 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-month treatment.

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

Footnotes

  • Funding information and disclosures are provided at the end of the article. Full disclosure form information provided by the authors is available with the full text of this article at Neurology.org/cp.

  • DMD Heart Study Group coinvestigators are listed in Appendix 2.

  • F. Muntoni and M. Guglieri are joint senior authors and contributed equally to the manuscript.

  • Study registration: DMD Heart Protection trial; EudraCT: 2007-005932-10 and ISRCTN 50395346.

  • Study Sponsor: NHS Hospitals Foundation Trust, UK (contact details: Newcastle Joint Research Office, Level 1 Regent Point, Gosforth, Newcastle upon Tyne NE3 3HD; Trust.R&D{at}nuth.nhs.uk). The study sponsor has not had any role in protocol design and/or in the reporting of findings, nor did it have ultimate authority over presentation of results.

  • Class of Evidence: NPub.org/coe

  • Received June 28, 2020.
  • Accepted November 6, 2020.
  • © 2021 American Academy of Neurology
View Full Text

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

REQUIREMENTS

You must ensure that your Disclosures have been updated within the previous six months. Please go to our Submission Site to add or update your Disclosure information.

Your co-authors must send a completed Publishing Agreement Form to Neurology Staff (not necessary for the lead/corresponding author as the form below will suffice) before you upload your comment.

If you are responding to a comment that was written about an article you originally authored:
You (and co-authors) do not need to fill out forms or check disclosures as author forms are still valid
and apply to letter.

Submission specifications:

  • Submissions must be < 200 words with < 5 references. Reference 1 must be the article on which you are commenting.
  • Submissions should not have more than 5 authors. (Exception: original author replies can include all original authors of the article)
  • Submit only on articles published within 6 months of issue date.
  • Do not be redundant. Read any comments already posted on the article prior to submission.
  • Submitted comments are subject to editing and editor review prior to posting.

More guidelines and information on Disputes & Debates

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. [email protected]
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 Publishing Agreement 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
    • Abstract
    • Methods
    • Results
    • Discussion
    • Acknowledgment
    • Study Funding
    • Disclosure
    • Appendix 1 Authors
    • Appendix 2 Coinvestigators
    • Footnotes
    • References
  • Figures & Data
  • Info & Disclosures
Advertisement

Hastening the Diagnosis of Amyotrophic Lateral Sclerosis

Dr. Brian Callaghan and Dr. Kellen Quigg

► Watch

Related Articles

  • No related articles found.

Alert Me

  • Alert me when eletters are published
Neurology: Clinical Practice: 14 (1)

Articles

  • Articles
  • 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: Education
  • Neurology: Genetics
  • Neurology: Neuroimmunology & Neuroinflammation
  • AAN.com
  • Continuum
  • Brain & Life
  • Neurology Today

Wolters Kluwer Logo

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

© 2023 American Academy of Neurology

  • Privacy Policy
  • Feedback
  • Advertise