RESUMEN
BACKGROUND: Current pediatric guidelines for heterozygous familial hypercholesterolemia (HeFH) propose pharmacotherapy (PT) with statins from age 8 to 10 years; however, schemes with absorption inhibitors combined with statins, could be started earlier. The aim of the study was to show the 10-year results of a combined treatment protocol. METHODS: Prospective, descriptive and analytical study. Pediatric patients (n=70; mean age at PT initiation 9.3 years [range, 2-17.5]) with HeFH who required PT between 2005 and 2015 were included. All patients ≥10 years, with LDL >190 mg/dL or >160 mg/dL with one cardiovascular risk factor (CVRF) or >130 mg/dL with two or more CVRF; and those patients 5-10 years and with LDL-C >240 mg/dL or a family history of a cardiovascular event before 40 years, were medicated. After a period on a lipid-lowering diet (LLD), all patients were started on ezetimibe. Patients who did not achieve the treatment goal were given statins. The variables were: age, age at PT initiation, duration of PT, initial LDL-C, mean LDL-C during ezetimibe monodrug therapy, mean LDL-C during combined PT, and percentage of LDL decrease. RESULTS: LDL-C levels were: Baseline: 235 mg/dL±55; after 3 months on ezetimibe: 167 mg/dL±47 (decrease: -27.62%). In 18 patients who did not reach the treatment goal atorvastatin was added and their LDL-C decreased -41.5% (p: 0.02). Overall, mean final LDL-C was 155 mg/dL±30.4 (range, 98-257) and treatment goals were reached in 74% of the patients. No severe side effects were reported. CONCLUSIONS: Combined and sequential treatment starting at early ages was shown to be safe and effective over this follow-up period.
Asunto(s)
Anticolesterolemiantes/uso terapéutico , Enfermedades Cardiovasculares/prevención & control , Ezetimiba/uso terapéutico , Inhibidores de Hidroximetilglutaril-CoA Reductasas/uso terapéutico , Hiperlipoproteinemia Tipo II/tratamiento farmacológico , Adolescente , Anticolesterolemiantes/efectos adversos , Argentina/epidemiología , Enfermedades Cardiovasculares/epidemiología , Enfermedades Cardiovasculares/etiología , Niño , LDL-Colesterol/sangre , Terapia Combinada/efectos adversos , Dieta con Restricción de Grasas/efectos adversos , Monitoreo de Drogas , Quimioterapia Combinada/efectos adversos , Ezetimiba/efectos adversos , Femenino , Estudios de Seguimiento , Hospitales Pediátricos , Humanos , Inhibidores de Hidroximetilglutaril-CoA Reductasas/efectos adversos , Hiperlipoproteinemia Tipo II/sangre , Hiperlipoproteinemia Tipo II/dietoterapia , Hiperlipoproteinemia Tipo II/fisiopatología , Lipoproteínas LDL/sangre , Masculino , Servicio Ambulatorio en Hospital , Guías de Práctica Clínica como Asunto , Estudios Prospectivos , Factores de Riesgo , Índice de Severidad de la EnfermedadRESUMEN
Familial hypercholesterolemia is one of the most common genetic disorders and it provides the best evidence on the etiologic role of LDL-colesterol for arteroesclerosis development. Two patients with severe hypercholesterolemia had been presented. Importance of early diagnosis and treatment has been stated to avoid or delay atherosclerosis and coronary heart disease.
Asunto(s)
Hipercolesterolemia , Niño , Femenino , Humanos , Hipercolesterolemia/diagnóstico , Hipercolesterolemia/genética , Hipercolesterolemia/terapia , Masculino , Linaje , Índice de Severidad de la EnfermedadRESUMEN
La hipercolesterolemia familiar es uno de los trastornos genéticos más comunes y aporta información sustancial sobre papel etiológico que el colesterol LDL tiene para el desarrollo de la ateroesclerosis. Se presentan dos pacientes con hipercolesterolemia grave. Se remarca la importancia del diagnóstico y tratamiento temprano para evitar o demorar la enfermedad ateromatosa y la enfermedad coronaria precoz.
Familial hypercholesterolemia is one of the most common genetic disorders and it provides the best evidence on the etiologic role of LDL-colesterol for arteroesclerosis development. Two patients with severe hypercholesterolemia had been presented. Importance of early diagnosis and treatment has been stated to avoid or delay atherosclerosis and coronary heart disease.
