Bourke, John P;
Bryant, Andrew;
Landon, Gregory;
Burn, Alexis;
Spinty, Stefan;
Quinlivan, Ros;
Alhaswani, Zoya;
... DMD Heart Study Group; + view all
(2025)
Preventing Cardiomyopathy in Duchenne Muscular Dystrophy: Long-Term Follow-Up of Patients in the Randomised, Placebo-Controlled Drug-Trial of Perindopril and Bisoprolol.
European Journal of Neurology
, 32
(3)
, Article e70097. 10.1111/ene.70097.
Preview |
Text
Heart Protection LTFU paper published.pdf - Published Version Download (393kB) | Preview |
Abstract
Introduction: It is uncertain whether using cardiac drugs prophylactically in combinations for DMD is better than ACE-inhibitor alone. Our previous study showed no differences in left ventricular function between perindopril-bisoprolol and matched placebo after 36 months. // Methods: This study aimed to determine whether heart measures diverged after 60-month total follow-up. All participants had commenced open-label perindopril and bisoprolol when the original study ended. All were reconsented for access to heart measures, undertaken as part of their clinical care. The primary outcome was the change in echo-measured ventricular ejection fraction from baseline according to original randomization. // Results: Of 75 participants reported originally, 65 (aged 16 ± 2.5 years) were re-recruited and had data for analysis. Adjusted primary outcomes included 44 participants (original arms: ‘active’ 21; ‘placebo’ 23), 48 for secondary outcomes, and 65 for ‘headcount’ analysis of those with ventricular dysfunction. Absolute LVEF% values reduced in both groups (‘active’: 62.5% ± 5.6% to 53.8% ± 4.0%; ‘placebo’: 60.6% ± 4.9% to 50.4% ± 8.5%). Despite trends favoring earlier introduction of therapy, change from baseline was similar between groups (adjusted mean difference: -7.7 (95% CI -16.4 to1.0%)). However, more in the ‘placebo’ arm had died, had reduced LVEF%, and were taking additional heart medications. // Conclusion: While some patients may have benefited from ‘early’ (active) as opposed to ‘delayed’ (placebo) initiation of perindopril and bisoprolol, group-mean ventricular function did not differ between study arms after 60 months. Small numbers, absence of a control group, insensitivity of echo-ejection fraction, and additional drug use probably prevented divergence between groups.
| Type: | Article |
|---|---|
| Title: | Preventing Cardiomyopathy in Duchenne Muscular Dystrophy: Long-Term Follow-Up of Patients in the Randomised, Placebo-Controlled Drug-Trial of Perindopril and Bisoprolol |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1111/ene.70097 |
| Publisher version: | https://doi.org/10.1111/ene.70097 |
| Language: | English |
| Additional information: | Copyright © 2025 The Author(s). European Journal of Neurology published by John Wiley & Sons Ltd on behalf of European Academy of Neurology. This is an open access article under the terms of the Creative Commons Attribution-NonCommercial License, https://creativecommons.org/licenses/by-nc/4.0/, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes. |
| Keywords: | ACE-inhibitor; beta-blocker; cardiomyopathy prevention; DMD; heart medications; prophylaxis |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Developmental Neurosciences Dept |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10206738 |
Archive Staff Only
 |
View Item |
