Re: A Systematic Review and Meta-Analysis of Statin Therapy in Children With Familial Hypercholesterolemia

I read this article. They admit that the optimal age to start statin
treatment in kids with heFH is unknown. They also say that "the
effect of lipid lowering treatment in childhood on CVD later in life"
is unclear. They call for more randomized clinical trials "on
(surrogate) clinical end points such as IMT and, ideally, on
cardiovascular events and death" in order to "clarify these issues."

It would have been interesting if they had specified what kind of
clinical trials they had in mind. In their meta-analysis, they only
included double-blind, randomized, placebo-controlled trials. A
placebo-controlled trial of statin treatment in heFH starting in
childhood and continuing for enough years that CVD events and
mortality could be used as endpoints would appear to be unethical,
even if it was otherwise practical. Even men with heFH don't usually
start having CVD events until their thirties or later (untreated).
So, presumably, that is not what they are proposing.

If they are thinking of a trial looking at statin treatment started at
different ages (e.g., age 10, 15, 20), that might be ethical but would
still have to be a very lengthy trial in order to use CVD events and
mortality as endpoints. It just doesn't seem practical to me.

I think the best we are going to get is a retrospective study
comparing kids with heFH who started statin treatment at different
ages (for whatever reason) and trying to control for the differences
between the groups. That won't be feasible until some years from now,
though. Such a study would also look at adverse events.

The state of current knowledge is that the benefits and risks of
starting statin treatment in childhood are both unknown. In the short
term (the longest trials so far have been 104 weeks) it appears to be
safe. In addition, the one double-blind, randomized, placebo-
controlled trial that used IMT as an endpoint found a benefit for the
treatment group. Whether starting statin treatment in childhood will
really make a difference in terms of reducing clinical events and
mortality in adulthood is just not known. My guess is that any
difference between groups where the age of treatment initiation
differs by just a few years (5 years, say) is going to be small, but
that's just my opinion.

As you would expect, the argument for starting treatment early is
stronger for men. The figure I recall seeing is that age of first CVD
event in heFH is 9 years earlier, on average, in men.

Marilyn

.

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