Effect of intensive LDL cholesterol lowering with PCSK9 monoclonal antibodies on tendon xanthoma regression in familial hypercholesterolemia. (August 2017) - Bea, Ana M.
Abstract: Background and aims: The effect of LDLc lowering with PCSK9 antibodies on tendon xanthomas (TX) is unknown. Methods: TX was measured in 24 heterozygous familial hypercholesterolemia (HeFH) cases and in 24 HeFH controls with or without PCSK9 inhibitors for at least one year. Results: Exposure to PCSK9 inhibitors in cases was 2.96 ± 1.33 years. LDLc decreased 80.8 ± 7.66% in cases and 56.9 ± 11.1% in controls. There was a decrease in maximum (−5.03%) and mean (−5.32%) TX in cases but not in controls (+3.97%, +3.16, respectively, p = 0.01). PCSK9 inhibitor treatment was independently associated with TX reduction. Conclusion: Addition of a PCSK9 inhibitor to statin and ezetimibe resulted in a greater decrease in LDLc and TX after 3 years of treatment. Highlights: This is the first study that assess the intensive LDL cholesterol lowering effect of PCSK9 inhibition on tendon xanthomas. Compared with standard lipid-lowering therapy, the combination with PCSK9 inhibitors showed greater tendon xanthoma regression. Tendon xanthoma regression was observed with mean LDL cholesterol of 60 mg/dL. Some patients developed asymptomatic calcifications inside the Achilles tendon. These results suggest that PCSK9 inhibition is highly efficient regressing lipid deposits in heterozygous familial hypercholesterolemia.