| Autor: |
Bijlani, Priyesha, Davidson, Michael, Duell, P.B., Horan, Mary, Malloy, Mary, Newfield, Ron, Pradeep, Pallavi, Shah, Prediman, Stock, Eveline, Warden, Bruce, Ito, Matthew, Wilkinson, Michael |
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| Zdroj: |
Journal of Clinical Lipidology; 2024 Supplement, Vol. 18 Issue 4, pe567-e568, 2p |
| Abstrakt: |
Homozygous familial hypercholesterolemia (HoFH) is an autosomal co-dominant disorder characterized by extreme elevations in LDL-C and early onset ASCVD. Unfortunately, patients with HoFH generally experience poor LDL-C lowering with standard lipid-lowering therapy and rarely achieve LDL-C goals. Evinacumab is a monoclonal antibody administered by monthly IV infusion that binds angiopoietin-like 3 and when added to standard lipid-lowering therapies lowers LDL-C by ∼50% in clinical trials involving patients with HoFH. Real-world effectiveness and safety of evinacumab is unknown. Examine the safety and efficacy of evinacumab in the real-world treatment of patients with HoFH across multiple US academic medical centers. We performed retrospective chart review to assess safety and efficacy of evinacumab in patients with HoFH in clinical practice at six US academic medical centers. The primary efficacy endpoint was the percent change in LDL-C from baseline to first follow-up and to most recent follow-up after evinacumab initiation. Secondary efficacy endpoints were percent change in non-HDL-C, triglycerides, total cholesterol, HDL-C, and achievement of an LDL-C <70 mg/dL. Adverse events were recorded. Twenty-four patients (mean age 38 yrs; range 5-75) with HoFH were followed for a median of 48 weeks. Fifty percent were female, 16 (66.7%) had ASCVD, 21 (87.5%) on a statin, 20 (83.3%) on ezetimibe, 16 (66.7%) on PCSK9i, 6 (24%) on lomitapide, and 8 (33.3%) undergoing lipoprotein apheresis (Table 1). Significant reductions in LDL-C, non-HDL-C, total cholesterol, triglycerides, and HDL-C were observed both at first follow-up (4 weeks) and most recent follow-up (48 weeks) (p < 0.001 for all). Significantly more patients achieved LDL-C <70 mg/dL after evinacumab was added (Table 2, Figure). No serious adverse events occurred and evinacumab was generally well-tolerated. 9 patients (37.5%) reported side-effects which were generally mild. Side-effects led to discontinuation of therapy in 1 patient due to fatigue and back pain. Across six US academic medical centers, evinacumab was generally well-tolerated by patients with HoFH and lowered LDL-C by ∼50%, consistent with results from clinical trials. [ABSTRACT FROM AUTHOR] |
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