British clinicians report the long-term results of a prophylactic treatment for cardiomyopathy in Duchenne muscular dystrophy (DMD):
- the combination of perindopril (a conversion enzyme inhibitor) and bisoprolol (a beta-blocker) had already been the focus of an initial randomised clinical trial by the same team in 2011 in children with DMD aged between 5 and 13 years,
- as the trial was inconclusive at the end of an initial 36-month phase, a longer cardiological follow-up (allowing a total of 60 months to elapse) was set up to try and see if there were any more conclusive differences,
- data from 44 of the 74 patients who took part in the pivotal phase were analysed,
- the benefit of combining the two products proved not to be statistically significant over the long term.
For a number of methodological reasons put forward by the authors, these apparently disappointing results do not call into question the international recommendations for cardioprotection in DMD.
