Treatment of pediatric heterozygous familial hypercholesterolemia 7 years after the EAS recommendations: Real-world results from a large French cohort - INRAE - Institut national de recherche pour l’agriculture, l’alimentation et l’environnement Access content directly
Journal Articles Archives de Pédiatrie Year : 2024

Treatment of pediatric heterozygous familial hypercholesterolemia 7 years after the EAS recommendations: Real-world results from a large French cohort

Patrick Tounian
Yann Pucheu
  • Function : Author

Abstract

ackground: Heterozygous familial hypercholesterolemia (HeFH) predisposes to premature cardiovascular diseases. Since 2015, the European Atherosclerosis Society has advocated initiation of statins at 8−10 years of age and a low-density lipoprotein cholesterol (LDL-C) target of <135 mg/dL. Longitudinal data from large databases on pharmacological management of pediatric HeFH are lacking. Objective: Here, we describe treatment patterns and LDL-C goal attainment in pediatric HeFH using longitudi- nal real-world data. Methods: This was a retrospective and prospective multicenter cohort study (2015−2021) of children with HeFH, diagnosed genetically or clinically, aged <18 years, and followed up in the National French Registry of FH (REFERCHOL). Data on the study population as well as treatment patterns and outcomes are summarized as mean§SD. Results: We analyzed the data of 674 HeFH children (age at last visit: 13.1 § 3.6 years; 82.0 % ≥10 years; 52.5 % females) who were followed up for a mean of 2.8 § 3.5 years. Initiation of lipid-lowering therapy was on average at 11.8 § 3.0 years of age for a duration of 2.5 § 2.8 years. At the last visit, among patients eligible for treatment (573), 36 % were not treated, 57.1 % received statins alone, 6.4 % statins with ezetimibe, and 0.2 % ezetimibe alone. LDL-C was 266§51 mg/dL before treatment and 147§54 mg/dL at the last visit (44.7 %) in treated patients. Regarding statins, 3.3 %, 65.1 %, and 31.6 % of patients received high-, moder- ate-, and low-intensity statins, respectively. Overall, 59 % of children on statin therapy alone and 35.1 % on bitherapy did not achieve the LDL-C goal; fewer patients in the older age group did not reach the treatment goal Conclusion: Pediatric patients with FH followed up in specialist lipid clinics in France receive late treatment, undertreatment, or suboptimal treatment and half of them do not reach the therapeutic LDL-C goal. Finding a more efficient framework for linking scientific evidence to clinical practice is needed
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hal-04539381 , version 1 (09-04-2024)

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Noel Peretti, Alexandre Vimont, Emmanuel Mas, Julie Lemale, Rachel Reynaud, et al.. Treatment of pediatric heterozygous familial hypercholesterolemia 7 years after the EAS recommendations: Real-world results from a large French cohort. Archives de Pédiatrie, 2024, 31 (3), pp.188-194. ⟨10.1016/j.arcped.2024.01.004⟩. ⟨hal-04539381⟩
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