Treating Pediatric FH: PCSK9 Inhibitors, Statins, and More


Daniel Gaudet, MD, PhD, discusses what's next in clinical pursuits of the greatly underdiagnosed hereditary cardiovascular disease.

New data presented in a late-breaking session at the European Society of Cardiology (ESC) 2020 Congress this weekend showed children with familial hypercholesterolemia (FH) reduced LDL-cholesterol (LDL-C) levels by 44.5% with PCSK9 inhibitor evolocumab (Repatha).

The findings from the phase 3 HAUSER study, presented by author Daniel Gaudet, MD, PhD, advanced the understanding of safely and effectively inhibiting PCSK9 in pediatric patients burdened with the common hereditary cardiovascular disease.

But the work into pediatric PCSK9 inhibition is far from done.

In an interview with HCPLive, Gaudet, a lipidologist and professor of Medicine at University of Montreal, discussed how the challenge of his team’s work is in assuring evolocumab is viable and beneficial in all eligible FH cases in children—not just over an extended period of time, but in all regions of the world.

Though many children and adolescents with the disease are adequately treated with statin therapy, he believes there will be a “considerable population” of FH cases in need of something like evolocumab—due to disease severity and early onset.

The early excitement of PCSK9 inhibition for LDL-C reduction and management is still fresh for the study author. Now, HAUSER shows its pediatric tolerability and efficacy.

“The real challenge of the next years is to continue to improve our knowledge of the safety and efficacy in larger cohorts,” Gaudet said. “And from a family approach: combining life habits, the use of statins, and then making sure that everywhere, in every continent, you have access to an accurate diagnosis, and an accurate treatment.”

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