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More than half of all children referred for heart transplants have chronic heart failure. Evidence based treatment is difficult because all the standard drugs for heart failure have been tested in adults only. The first ever randomised trial in children compared the β blocker carvedilol with placebo in 161 children and adolescents with heart failure complicating dilated cardiomyopathy or congenital heart disease. It didn't work. Children given the drug in addition to their other drugs for heart failure were just as likely to get worse as children given a placebo (25/103 (24%) v 16/54 (30%); odds ratio 0.79, 95% CI 0.36 to 1.59).
The result was surprising—because β blockers work so well in adults—and disappointing. It is possible that the trial was too small to find a clinically relevant difference between the groups, say the authors. Or perhaps the outcome they used wasn't sensitive enough, the dose of carvedilol wasn't big enough, or the children were too heterogeneous.
Alternatively, β blockers may work differently on young hearts that fail from aetiologies other than ischaemia. Children are not simply small adults, says an editorial (p 1214). This trial is an important illustration of the limitations of using adult evidence in children.