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Background: 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 longitudinal 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-, moderate-, 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., Competing Interests: Declaration of competing interest • NP has received honoraria from ADEN-ALLP, Alexion AMGEN, Amryt, Biocodex, Elsevier, Kowa, Lactalis, Nestlé NHS, NHC, Nutricia-Danone, Orphan Europ, Sanofi, Shire, Ultragenyx • PT has participated in one scientific committee at Ultragenyx laboratories • YP has received honoraria from Servier, Viatris, AMGEN, Sanofi, Bouchara-Recordati • JPR has received remuneration from Sanofi, Ultragenyx • EB has received honoraria from AstraZeneca, AMGEN, MSD, Sanofi and Regeneron, Aegerion/Amryt, Lilly, Ionis-pharmaceuticals, Akcea, Alexion pharma, Servier, Novartis, Mylan/Viatris, Organon and Genfit • AG reports grants and personal fees from AMGEN, Sanofi, Regeneron, Mylan Viatris, MSD, Akcea Therapeutics, Amryt and Ultragenyx. • SB has received honoraria for board, conferences, clinical trial or congress from Aegerion, Akcea, Elivie, Novartis, Sanofi or AMGEN • PP, LR, AV, RR have no conflict of interest related to this article., (Copyright © 2024 French Society of Pediatrics. Published by Elsevier Masson SAS. All rights reserved.)