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Efficacy and Safety of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia: Phase 3, Multinational Open-Label Study.

Author: Baccara-DinetMarie T, BruckertEric, CaprioSonia, CharngMin-Ji, DanielsStephen R, ManvelianGaren, OurliacAnne, ScemamaMichel, WiegmanAlbert, Zárate-MoralesCézar A

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Clinical Trial, Phase IIIA Phase III trial is a trial that further rigorously verifies the efficacy and safety of a new drug or treatment, whose efficacy and safety have been confirmed in a Phase II trial, in a large number of patients, and evaluates whether it is superior to existing treatments.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Overview

The safety and effectiveness of alirocumab, a PCSK9 inhibitor, were evaluated in pediatric patients with homozygous familial hypercholesterolemia who had not achieved desired cholesterol levels.
Paper Details　
Original Abstract of the Article :
Despite progress in treating homozygous familial hypercholesterolemia, most patients do not achieve low-density lipoprotein cholesterol (LDL-C) targets. This study examined efficacy and safety of the PCSK9 (proprotein convertase subtilisin/kexin type 9) inhibitor, alirocumab, in pediatric patients (...See full text at original site
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引用元：
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9750107/

データ提供：米国国立医学図書館（NLM）

A New Horizon in Homozygous Familial Hypercholesterolemia: Alirocumab Shines in Pediatric Patients

Homozygous familial hypercholesterolemia (HoFH) is a rare and challenging condition, often leading to high levels of low-density lipoprotein cholesterol (LDL-C). This study ventures into the vast desert of HoFH, investigating the efficacy and safety of alirocumab, a PCSK9 inhibitor, in pediatric patients. The researchers conducted a phase 3 open-label study, demonstrating the efficacy of alirocumab in reducing LDL-C levels and its favorable safety profile in pediatric patients with HoFH.

Alirocumab: A Beacon of Hope in the Desert of HoFH

The study highlights the efficacy and safety of alirocumab in treating HoFH in pediatric patients. This research is a beacon of hope in the desert of HoFH, offering a promising treatment option for children and adolescents living with this condition.

Navigating the Desert of HoFH: A Compass for Pediatric Care

The study underscores the importance of understanding the specific needs of pediatric patients with HoFH and highlights the potential of alirocumab as a valuable treatment option. This research provides valuable insights for healthcare providers, guiding them in their efforts to manage HoFH in children and adolescents.

Dr. Camel's Conclusion

This study demonstrates the efficacy and safety of alirocumab in treating HoFH in pediatric patients. This research is a significant contribution to the field of pediatric cardiology, offering a promising treatment option for children and adolescents with HoFH.

Date :
  1. Date Completed 2022-11-29
  2. Date Revised 2023-12-02
Further Info :

Pubmed ID

36325897

DOI: Digital Object Identifier

PMC9750107

SNS
PICO Info
in preparation
Languages

English

Positive IndicatorAn AI analysis index that serves as a benchmark for how positive the results of the study are. Note that it is a benchmark and requires careful interpretation and consideration of different perspectives.

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