Treating children with familial hypercholesterolemia (FH) early can help reduce progression of atherosclerosis without adverse events related to growth and development, said Raul Santos, MD, PhD, director, Lipid Clinic at the Heart Institute, and associate professor, University of São Paulo Medical School, Brazil.
Treating children with familial hypercholesterolemia (FH) early can help reduce progression of atherosclerosis without adverse events related to growth and development, said Raul Santos, MD, PhD, director, Lipid Clinic at the Heart Institute. and associate professor, University of São Paulo Medical School, Brazil.
Why has early treatment of familial hypercholesterolemia with therapy been controversial?
You know, there's a lot of discussion why we should start treating kids early, right? Because usually atherosclerosis, it starts developing very early, but manifestation will be, let's say, by the age of 40, 45, 50. So, people say, “Oh, maybe you can wait for these kids to become adults and then we could start treating them.” But, actually, we have evidence that if you start treating early, like let's say, the age of 10 or even earlier, we can reduce the progression of atherosclerosis. We can use imaging techniques to show that the disease progression is much, much lower. And actually, we can even regress that. So this is one argument in favor of treating early.
Second, now we have—at least with the statins—20 years of follow-up showing the drugs are safe and they don't compromise growth, sexual development, cognitive function, and everything. And by the way, I think it's important to say that, actually, evolocumab was very safe regarding growth, sexual development, steroid hormones, lack of soluble vitamins. So, actually, we measured all of that and in the kids it was great. And with the statins, it’s the same thing. So, I think it's time to stop this controversy and start, of course, if we understand that those kids have enough risk, very high [low-density lipoprotein cholesterol], bad family history, start treatment as soon as possible.
What are the benefits and risks of early treatment?
You know, actually, lipid-lowering therapy for kids is very safe. There might be some concern that because kids might need cholesterol for growing up, for brain development, there might be some trouble with that, but this is not true. Actually, we saw in in HAUSER, we didn't see any adverse events related to growth, pubertal development…neurocognitive function. And actually, we also had seen with the statins, the same thing. So, actually, even if we put, let's say, a very powerful therapy on top of drugs that kids are already taking, we didn't see anything that is bad. Actually, we have follow-ups up to 2 years. Of course, we say 2 years is not a lifetime, but as I said, we have more than 20 years follow-up with the statins and we didn’t see any harm.