ArticleJournal of the American Heart Association2023
Real-World Effectiveness of PCSK9 Inhibitors in Reducing LDL-C in Patients With Familial Hypercholesterolemia in Italy: A Retrospective Cohort Study Based on the AIFA Monitoring Registries.
Article in Journal of the American Heart Association, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
23 citing papers in PubMed, 1 synthesis or guideline pooled it, 27 citations in OpenAlex.
- Efficacy and safety of Ongericimab in patients with hypercholesterolemia: a systematic review and meta-analysis.European journal of clinical pharmacology · 2026Pooled it
- Comparative effectiveness and safety of inclisiran versus evolocumab and alirocumab: a 180-day real-world study.BMC cardiovascular disorders · 2026Article
- High-capacity adenoviral vector-mediated expression of an LDLR/transferrin chimeric protein in muscle reduces atherosclerosis in LdlrMolecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Adherence to PCSK9 Inhibitors in Clinical Practice: Systematic Review and Meta-Analysis of Observational Studies.JACC. Advances · 2026Article
- PCSK9 Inhibitors Reduce Oxidative Stress Biomarkers in Heterozygous Familial Hypercholesterolemia.Journal of cellular and molecular medicine · 2026Article
- Non-Responder to Inclisiran and Evolocumab-A Female Patient with Heterozygous Familial Hypercholesterolemia and Statin Intolerance.Diseases (Basel, Switzerland) · 2026Article
- PCSK9 inhibitors patterns of use in France from nationwide repeated cross-sectional and cohort studies.Scientific reports · 2026Article
- Treatment pathways of lipid-lowering therapies in Germany 2016-2022.Clinical research in cardiology : official journal of the German Cardiac Society · 2026Article
- Biochemical characterisation of familial hypercholesterolemia: Associations between genetic and lipid profiles.Journal of medical biochemistry · 2026Article
- Translating the effect of dual lipid reduction with PCSK9 inhibitors on a mechanical vascular instrumental biomarker in familial hypercholesterolemia subjects.Journal of translational medicine · 2025Observational
- Impact of PCSK9 Inhibitors on Lipoprotein(a) Levels: A Multi-Center Study.European journal of preventive cardiology · 2025Article
- A Comprehensive Review of the Latest Approaches to Managing Hypercholesterolemia: A Comparative Analysis of Conventional and Novel Treatments: Part II.Pharmaceuticals (Basel, Switzerland) · 2025Review
- Genomic and Precision Medicine Approaches in Atherosclerotic Cardiovascular Disease: From Risk Prediction to Therapy-A Review.Biomedicines · 2025Review
- Pediatrics cascade screening in inherited dyslipidemias: a lipoprotein apheresis center experience.Endocrine · 2025Article
- A therapeutic cooperation by bempedoic acid and proprotein convertase subtilisin/kexin type 9 inhibitors in statin intolerance inherited dyslipidemias.Internal and emergency medicine · 2025Article
- Quo Vadis after AEGIS: New Opportunities for Therapies Targeted at Reverse Cholesterol Transport?Current atherosclerosis reports · 2025Review
- Emerging Biomarkers and Determinants of Lipoprotein Profiles to Predict CVD Risk: Implications for Precision Nutrition.Nutrients · 2024Review
- Monoclonal Anti-PCSK9 Antibodies: Real-World Data.Journal of clinical medicine · 2024Article
- Recurrent Acute Coronary Syndrome in Young Man with Familial Hypercholesterolemia: Efficacy of Evolocumab Add-On Treatment Demonstrated via Serial Coronary Angiography.Biomedicines · 2024Article
- Targeting Allosteric Site of PCSK9 Enzyme for the Identification of Small Molecule Inhibitors: An In Silico Drug Repurposing Study.Biomedicines · 2024Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors at 5 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Background Information on the real-world use of proprotein convertase subtilisin kexin 9 inhibitors (PCKS9is) in familial hypercholesterolemia are limited. We evaluated the pattern of prescription and the long-term efficacy of alirocumab and evolocumab in Italian patients with familial hypercholesterolemia in clinical practice. Methods and Results The data set for analysis was extracted from the PCKS9i Italian Medicines Agency (AIFA) registry and included 2484 patients with heterozygous familial hypercholesterolemia (HeFH) and 62 patients with homozygous familial hypercholesterolemia (HoFH) who were prescribed PCKS9is from February 2017 to December 2021. As the follow-up schedules were not prespecified and could vary, persistence and adherence as well as low-density lipoprotein cholesterol (LDL-C) changes during 2 years of treatment were analyzed in a final cohort of 1299 patients with familial hypercholesterolemia. At baseline, 53.8% of patients with HeFH and 69.4% of patients with HoFH were receiving maximally tolerated lipid-lowering therapies, while 45.9% of patients with HeFH and 30.7% of patients with HoFH reported statin intolerance; mean LDL-C was 197.7±52.3 mg/dL in HeFH and 252.0±106.2 mg/dL in HoFH. The 6-month persistence and adherence to therapy were >85%, and LDL-C reduction reached 58.6% (to 79.7 mg/dL) in HeFH and 57.6% (to 95.1 mg/dL) in HoFH after 24 months of treatment. The European Atherosclerosis Society/European Society of Cardiology LDL-C goals were achieved in 43.3% of patients with HeFH and 37.5% of patients with HoFH. Conclusions PCKS9i prescribed to patients with familial hypercholesterolemia in clinical practice showed LDL-C-lowering efficacy similar to that observed in controlled trials. However, 2 of 5 HeFH cases and 2 of 6 HoFH cases achieved the recommended LDL-C goals. The full achievement of European Atherosclerosis Society/European Society of Cardiology LDL-C goals should require a lower threshold for PCKS9i initiation and a combination of multiple therapies.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.