RESUMEN
BACKGROUND: In Poland, treatment with proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors has become available free of charge in a therapeutic program. Assessed herein, is the efficacy and safety of alirocumab and evolocumab in patients with heterozygous familial hypercholesterolemia (FH). METHODS: Data of 55 adult FH patients who participated in the program were analyzed upon meeting the criteria established by the Ministry of Health (low density lipoprotein cholesterol [LDL-C] above 160 mg/dL on max. tolerated statin dose and ezetimib). The efficacy of PCSK9 inhibitors in reducing LDL-C with drug administration every 2 weeks was assessed after 3 months and 1 year of therapy. A safety profile evaluation was performed at each visit. 48 patients completed the 3-month and 21 for the 1-year observation periods (34 patients treated with alirokumab and 14 with evolocumab). RESULTS: The mean concentration of direct-measured LDL-C decreased from the initial level of 215.1 ± 74.5 mg/dL to 75.3 ± 64.1 mg/dL, i.e., by 65 ± 14% following 3 months of treatment. This effect was stable in 1-year observation (77.7 ± 72.8 mg/dL). Adverse effects were flu-like symptoms (13.0%), injection site reactions (11.1%), fatigue (5.6%) and musculoskeletal symptoms (5.6%). Seven patients failed to complete the 3-month treatment period due to side effects or non-compliance, and 1 patient failed to complete the 1-year treatment due to myalgia. CONCLUSIONS: This study confirmed high effectiveness of PCSK9 inhibitors in reducing LDL-C levels in patients with FH. Due to restrictive inclusion criteria with LDL-C threshold level > 160 mg/dL (> 4.1 mmol/L) required for participation in the therapeutic program, a relatively small number of FH patients were eligible for treatment.
Asunto(s)
Hiperlipoproteinemia Tipo II , Proproteína Convertasa 9 , Adulto , Anticuerpos Monoclonales/efectos adversos , LDL-Colesterol , Humanos , Hiperlipoproteinemia Tipo II/diagnóstico , Hiperlipoproteinemia Tipo II/tratamiento farmacológico , Inhibidores de PCSK9 , Polonia , Resultado del TratamientoRESUMEN
BACKGROUND: The 2014 ACC/AHA guidelines redefined the strategy for LDL-cholesterol (LDL-C) treatment. According to data from evidence-based studies, the basis for earlier therapeutic recommendations for LDL-C target levels (2.6 and 1.8 mmol/L) may be disputed, and only the data for the percent LDL-C reduction are objective. The target is a moderate intensity (30-50%) LDL-C reduction in the high cardiovascular (CV) risk group, and a high intensity LDL-C reduction (> 50%) in the very high risk group. In our study, we analysed the success of the two types of strategies in attaining the target in the everyday routine. METHODS: Of 5652 patients suffering from hypercholesterolemia, 4302 underwent treat-to-target treatment, and 1350 patients were treated with a percent reduction strategy. Physicians were free to choose the dosage and the target treatment form. The 12-month study included three follow-up visits. RESULTS: In the high CV risk, statin-naive subgroup the percent LDL-C reduction strategy has been proven to be clearly more successful than the treat-to-target strategy, i.e. a higher proportion of patients reached the target values. We observed that the absolute value corresponding to a percent reduction target is higher if the baseline LDL-C is higher, and therefore it is easier to reach. CONCLUSION: Therefore, in this large subgroup of patients with baseline LDL-C level higher than 3.9 mmol/L may be recommended the adaptation of the percent reduction assessment.