Endocrine Abstracts

Introduction: Recently, PCSK9 inhibitors have been approved in our country for familial hypercholesterolemia and for patients with cardiovascular diseases. They are still scarce data in real life patients effects.

Objective: Analyse the features of first patients treated with PCSK9 inhibitors in a specific unit of familial dyslipidemia and the effect on lipid profile and other clinical variables.

Material and methods: Data from patients with familial hypercholesterolemia treated with PCSK9 inhibitors were collected. Clinical data, physical examination and blood test data were collected at baseline, 3 months, 1 and 2 years.

Results: Data were obtained from 35 patients, 50% male. Mean age: 49.5 years (26–74). Mean follow-up: 12 years. Diagnosis: 11.4% combined familial hyperlipemia and 82.9% heterocigous familial hypercholesterolemia, 43% cases with mutation detected in LDL-R. 71% had a family history of premature CVD, 28.6% with obesity, diabetes 19.2%, smokers 2.9%, HTA 43,8%, 71.4% with high lipoprotein(a), 42.4% with early CVD (71% AMI). 69.6% treated with statins (54.3% rosuvastatin, 2.9% atorvastatin, 5.7% Fluvastatina), 66.7% ezetimibe, 20% resincolestiramina. 23% had elevated transaminases with at least two statins and 54.3% did not tolerate adequate dose of statin/ezetimibe because of secondary effects. The mean pre and postreatment levels (3–6 months) were: TC: 246.5±51.6 vs 147.5±39.4 mg/dl (P 0.001), LDL-C: 160.2±42.5 vs 70.3±28.6 (P 0.001), 60% achieved therapeutic objective, HDL-C: 55.4±17.9 vs 51.1±14.5 (P 0.16), non HDL-C:194.3±31.5 vs 97.8±30.7 (P 0.001), TG:158.1±46.2 vs 128±56.8 (P 0.25), Apo B 135.8±22.9 vs 78.3±17.7 (P 0.01), lipoprotein (a)98.2±54.9 vs 77.5±40.1 (P 0.006). There were no significant changes in: Blood pressure, heart rate, HbA1c, thyroid hormones, cortisol, Vitamin D, creatinine, transaminases and cell blood counts. Weight was 75.4±14.2 vs 73.6±13.4 (P 0.039) and testosterone 5.06±0.93 vs 4.2±1.1 (0.043) After 2 years of treatment: mean TC was 149.5±49.4, LDL: 75.3±34.6, HDL 54.1±17.5, nonHDL 96.3±38.2, TG 118.1±41.1, Apo B:47±10.6, Lipoprotein (a): 85.8±59.09 (non stadistical differences with effects at 3–6 months) There were no stadistical differences between alirocumab and evolocumab effects. Only four patients had mild side effect: 3 patients with pseudogripal syndrome and another patient with injection site reaction.

Conclusion: PCSK9 inhibitors were well tolerated and significantly reduced levels of TC, LDL-C, ApoB, non-HDL C and lipoprotein (a) after 2 years of treatment, with 60% of patients achieving therapeutic goals on LDL.