Effective treatment of high cholesterol in children and teens: A Synthesis of Findings from 24 Studies

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Original Abstract of the Article

Key Research Findings

Research on effective treatment of high cholesterol in children and teens is yielding promising results, suggesting both efficacy and safety. These studies evaluate a variety of treatments and identify potential strategies for managing high cholesterol in this population. For instance, 2 suggests that lifestyle modifications like exercise and weight loss can help lower blood pressure in adults with mild hypertension. Similarly, 18 reports that a national survey conducted by the LIPIGEN Paediatric Group in Italy recommends dietary modifications and statins as essential elements in managing children and teens diagnosed with familial hypercholesterolemia (FH). These studies highlight the multifaceted nature of high cholesterol management in children and teens, emphasizing the importance of personalized treatment plans.

Treatment Summary

2 recommends lifestyle modifications, including exercise and weight loss, to lower blood pressure in individuals with mild hypertension. 18 reports that the LIPIGEN Paediatric Group in Italy advocates for dietary interventions and statins as primary treatments for children and teens diagnosed with familial hypercholesterolemia (FH). 19 reviews algorithms for treating dyslipidemia in youth and proposes enhanced algorithms to address potential shortcomings. 24 presents a phase 3, multinational open-label study examining the efficacy and safety of the PCSK9 inhibitor alirocumab for children and adolescents with inadequately controlled homozygous familial hypercholesterolemia. Finally, 16 evaluates the efficacy and safety of evinacumab, a novel LDLR-independent lipid-lowering therapy, in pediatric patients with HoFH from parts A and B of a 3-part study.

Benefits and Risks

Benefits Summary

Treating high cholesterol in children and teens can offer several benefits, including improved blood pressure management, controlled lipid levels, and reduced risk of heart disease. Lifestyle modifications like exercise and weight loss have been shown to effectively lower blood pressure, as illustrated in 2 . Moreover, 18 demonstrates the efficacy of dietary modifications and statins in managing FH in this population. These treatments play a crucial role in maintaining cardiovascular health in children and teens.

Risks Summary

While treating high cholesterol in children and teens offers benefits, there are potential risks associated with treatment methods. Medications, for instance, can lead to side effects such as muscle aches, gastrointestinal issues, and liver abnormalities. 24 highlights the risk of injection site reactions and headaches with alirocumab treatment. It is essential to weigh the benefits and risks carefully when choosing treatment options for high cholesterol in children and teens.

Comparison of Studies

Similarities

Studies investigating high cholesterol management in children and teens share the common theme of demonstrating the potential benefits of lifestyle modifications and medication in controlling lipid levels and reducing cardiovascular risks. These studies underscore the need for a multifaceted approach to treating high cholesterol in this population.

Differences

Studies examining high cholesterol management in children and teens differ in their target age groups, treatment methods, and evaluation criteria. For example, 2 focuses on adults with mild hypertension, while 18 investigates children and teens with familial hypercholesterolemia (FH). These distinctions highlight the potential for tailored treatment strategies depending on age and severity of the condition.

Consistency and Inconsistencies of Results

Studies investigating high cholesterol management in children and teens often show consistent results. However, certain studies present some inconsistencies. For example, 24 demonstrates the effectiveness of alirocumab, while other studies may indicate potential risks associated with side effects. These discrepancies might be attributed to differences in study design and patient populations.

Practical Application and Considerations

Managing high cholesterol in children and teens demands a personalized approach. When applying these research findings to real-life situations, factors such as the child's age, severity of the condition, and lifestyle must be considered. Following medical advice and undergoing regular check-ups can maximize treatment efficacy and minimize the risk of side effects.

Limitations of Current Research

The current body of research on high cholesterol management in children and teens is still developing. More research is needed to assess long-term efficacy and safety. Studies involving diverse patient populations with varying ages and severities of the condition are crucial for developing more effective treatments. Additionally, understanding the impact of these treatments on a child's growth and development requires further investigation.

Future Research Directions

Future research on high cholesterol management in children and teens should focus on long-term efficacy and safety assessments. Conducting studies with diverse patient populations, considering different ages and severities of the condition, is vital for developing more effective treatments. Moreover, investigating the impact of these treatments on a child's growth and development is crucial.

Conclusion

Treating high cholesterol in children and teens offers promising benefits, including improved blood pressure control, reduced lipid levels, and lower risk of heart disease. These treatments play a critical role in maintaining cardiovascular health in this population. However, it is essential to carefully weigh the benefits and risks when selecting treatment options. Following medical advice and undergoing regular check-ups can maximize treatment efficacy and minimize the risk of side effects. Continued research will refine treatment strategies and address any unanswered questions regarding long-term outcomes and the impact of these treatments on a child's development.

Treatment Summary

The following treatment strategies are commonly employed for managing high cholesterol in children and teens:

  • Lifestyle modifications (exercise, weight loss)
  • Dietary interventions
  • Statins
  • PCSK9 inhibitors (Alirocumab, Evinacumab)
Literature analysis of 24 papers
Positive Content
21
Neutral Content
2
Negative Content
1
Article Type
5
9
8
10
23
1.

Effect of antihypertensive treatment in patients having already suffered from stroke. Gathering the evidence. The INDANA (INdividual Data ANalysis of Antihypertensive intervention trials) Project Collaborators.

Author: GueyffierF, BoisselJ P, BoutitieF, PocockS, CoopeJ, CutlerJ, EkbomT, FagardR, FriedmanL, KerlikowskeK, PerryM, PrineasR, SchronE

Reduced cardiovascular risk

Treating high blood pressure has been shown to reduce the risk of cardiovascular problems. Stroke, which can cause death or disability, is particularly linked to high blood pressure and is among the most preventable complications. People who have already had a stroke are at higher risk of having another one. Past studies on lowering blood pressure in stroke survivors have had inconclusive results.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Meta-AnalysisA statistical technique that integrates the results of multiple studies to draw more general conclusions about a particular issue.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

2.

Exercise and weight loss reduce blood pressure in men and women with mild hypertension: effects on cardiovascular, metabolic, and hemodynamic functioning.

Author: BlumenthalJ A, SherwoodA, GulletteE C, BabyakM, WaughR, GeorgiadesA, CraigheadL W, TweedyD, FeinglosM, AppelbaumM, HayanoJ, HinderliterA

Improved metabolic functioning
Increased cardiac output
Reduced blood pressure
Reduced peripheral resistance

Aerobic exercise combined with weight loss is recommended for the management of elevated BP in sedentary, overweight individuals.

Clinical TrialA clinical trial is a planned study conducted on humans to evaluate the efficacy and safety of pharmaceuticals, medical devices, and treatment methods. It aims to explore the possibilities of new treatments and improve the health of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, U.S. Gov't, P.H.S.Research funding provided by the Public Health Service (PHS).

Language : English

3.

Interventions used to improve control of blood pressure in patients with hypertension.

Author: FaheyT, SchroederK, EbrahimS

Hypertension treatment
Uncontrolled hypertension

Many people with high blood pressure (hypertension) don't reach their treatment goals, a situation known as uncontrolled hypertension. There's no clear consensus on the best way to organize and deliver care to help these patients reach their goals.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Meta-AnalysisA statistical technique that integrates the results of multiple studies to draw more general conclusions about a particular issue.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

4.

Interventions used to improve control of blood pressure in patients with hypertension.

Author: FaheyT, SchroederK, EbrahimS

Improved hypertension treatment
Hypertension

Many people with high blood pressure don't reach their treatment goals, a situation called uncontrolled hypertension. There's no clear agreement on the best way to organize and deliver care to help these patients achieve their goals.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Meta-AnalysisA statistical technique that integrates the results of multiple studies to draw more general conclusions about a particular issue.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

5.

Drugs for treatment of very high blood pressure during pregnancy.

Author: DuleyL, Henderson-SmartD J, MeherS

Fewer side effects
Lower persistent high blood pressure
Caesarean section
Higher risk of eclampsia
Higher risk of persistent high blood pressure
Hypotension
Respiratory difficulties

The choice of antihypertensive drug for very high blood pressure during pregnancy should depend on the clinician's experience and familiarity with the drug. Diazoxide, ketanserin, nimodipine, and magnesium sulfate should probably be avoided.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Meta-AnalysisA statistical technique that integrates the results of multiple studies to draw more general conclusions about a particular issue.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

6.

Interventions used to improve control of blood pressure in patients with hypertension.

Author: FaheyT, SchroederK, EbrahimS

Improved hypertension treatment
Hypertension

Many people with high blood pressure don't reach their treatment goals, a situation known as uncontrolled hypertension. There's no clear consensus on the best way to organize and deliver care to help these patients reach their goals.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Meta-AnalysisA statistical technique that integrates the results of multiple studies to draw more general conclusions about a particular issue.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

7.

A multifaceted intervention to improve blood pressure control: The Guideline Adherence for Heart Health (GLAD) study.

Author: BondsDenise E, HoganPatricia E, BertoniAlain G, ChenHaiying, ClinchC Randall, HiottAnn E, RosenbergerErica L, GoffDavid C

Improved BP control

High blood pressure is a major cause of illness and death, but many people don't reach their blood pressure goals. This study examines the effects of a multi-faceted intervention aimed at improving how doctors follow high blood pressure guidelines in primary care settings.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.

Language : English

8.

Interventions used to improve control of blood pressure in patients with hypertension.

Author: GlynnLiam G, MurphyAndrew W, SmithSusan M, SchroederKnut, FaheyTom

Improved blood pressure control
Lower mortality

An organized system of regular review and vigorous antihypertensive drug therapy reduced blood pressure and mortality. Self-monitoring, educational interventions, and appointment reminder systems may be useful adjuncts to improve blood pressure control.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Meta-AnalysisA statistical technique that integrates the results of multiple studies to draw more general conclusions about a particular issue.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

9.

Self-monitoring and other non-pharmacological interventions to improve the management of hypertension in primary care: a systematic review.

Author: GlynnLiam G, MurphyAndrew W, SmithSusan M, SchroederKnut, FaheyTom

Reduced blood pressure
Reduced mortality

This systematic review found that self-monitoring was associated with net reductions in systolic and diastolic blood pressure in patients with hypertension. An organised system of regular review allied to vigorous antihypertensive drug therapy was shown to reduce blood pressure and all-cause mortality.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Meta-AnalysisA statistical technique that integrates the results of multiple studies to draw more general conclusions about a particular issue.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

10.

Combination therapy as initial treatment for newly diagnosed hypertension.

Author: ByrdJames B, ZengChan, TavelHeather M, MagidDavid J, O'ConnorPatrick J, MargolisKaren L, SelbyJoe V, HoP Michael

Blood pressure control

Guidelines recommend using multiple medications to increase the chances of controlling high blood pressure. However, we lack information on how often doctors prescribe combinations of blood pressure medications when starting new treatment for hypertension.

Comparative StudyResearch that compares multiple groups/interventions to determine differences/similarities in outcomes/characteristics.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, N.I.H., ExtramuralResearch funding provided by the NIH to external research institutions and researchers.
Research Support, U.S. Gov't, Non-P.H.S.Research funding provided by US government agencies other than the Public Health Service.

Language : English

11.

Drugs for treatment of very high blood pressure during pregnancy.

Author: DuleyLelia, MeherShireen, JonesLeanne

Blood pressure reduction

Very high blood pressure during pregnancy can be dangerous for both mother and baby. While medications are used to lower blood pressure quickly and safely, there's uncertainty about which medications work best, their safety, and how they affect other pregnancy outcomes.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Meta-AnalysisA statistical technique that integrates the results of multiple studies to draw more general conclusions about a particular issue.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

12.

Association of Blood Pressure Lowering With Mortality and Cardiovascular Disease Across Blood Pressure Levels: A Systematic Review and Meta-analysis.

Author: BrunströmMattias, CarlbergBo

Reduced CVD risk
Reduced death risk
BP level dependence

Lowering blood pressure (BP) is associated with a reduced risk of death and cardiovascular disease (CVD) in individuals with baseline systolic BP (SBP) of 140 mm Hg or higher. This benefit was most pronounced in those with SBP of 160 mm Hg or above, where treatment was associated with a substantial reduction in major cardiovascular events. However, at lower BP levels, treatment was not associated with any benefit in primary prevention. In patients with previous coronary heart disease (CHD), treatment was associated with a reduced risk of major cardiovascular events, but not with survival.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Meta-AnalysisA statistical technique that integrates the results of multiple studies to draw more general conclusions about a particular issue.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

13.

Effectiveness of a multifactorial intervention, consisting of self-management of antihypertensive medication, self-measurement of blood pressure, hypocaloric and low sodium diet, and physical exercise, in patients with uncontrolled hypertension taking 2 or more antihypertensive drugs: The MEDICHY study.

Author: Unda VillafuerteFabián, Llobera CànavesJoan, Lorente MontalvoPatricia, Moreno SanchoMaría Lucía, Oliver OliverBartolomé, Bassante FloresPatricia, Estela MantolanAndreu, Pou BordoyJoan, Rodríguez RuizTomás, Requena HernándezAna, LeivaAlfonso, Torrent QuetglasMatíes, Coll BenejamJosé María, D'Agosto FortezaPilar, Rigo CarratalàFernando,

Hypertension treatment

High blood pressure is a major risk factor for heart disease, leading to serious health problems and death. While medication can help control blood pressure, less than half of people with high blood pressure reach their target. This is due to factors like delayed treatment, not taking medication as prescribed, and unhealthy habits (like eating too much fat and salt, not exercising enough, and being overweight). People with high blood pressure who are on medication but whose blood pressure isn't well-controlled have a higher risk of heart disease and death.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.

Language : English

14.

Clinical practice recommendations on lipoprotein apheresis for children with homozygous familial hypercholesterolemia: an expert consensus statement from ERKNet and ESPN.

Author: ReijmanM Doortje, KustersD Meeike, GroothoffJaap W, ArbeiterKlaus, DannEldad J, de BoerLotte M, de FerrantiSarah D, GalloAntonio, Greber-PlatzerSusanne, HartzJacob, HudginsLisa C, IbarretxeDaiana, KayikciogluMeral, KlingelReinhard, KolovouGenovefa D, OhJun, PlankenR Nils, StefanuttiClaudia, TaylanChristina, WiegmanAlbert, SchmittClaus Peter

Cardiovascular disease prevention
LDL-C reduction

This consensus statement on lipoprotein apheresis (LA) in children with homozygous familial hypercholesterolaemia (HoFH) provides guidance on its indication, methods, treatment targets, and follow-up. LA is an effective treatment for HoFH, but more research is needed to develop evidence-based guidelines for pediatric use.

PreprintAn early version of a research paper before it is peer-reviewed. It aims to share research with the community early on.

Language : English

16.

Evinacumab for Pediatric Patients With Homozygous Familial Hypercholesterolemia.

Author: WiegmanAlbert, Greber-PlatzerSusanne, AliShazia, Doortje ReijmanM, BrintonEliot A, CharngMin-Ji, SrinivasanShubha, Baker-SmithCarissa, BaumSeth, BrothersJulie A, HartzJacob, MoriartyPatrick M, MendellJeanne, BihorelSébastien, BanerjeePoulabi, GeorgeRichard T, HirshbergBoaz, PordyRobert

Lipid-lowering therapy for HoFH

This study evaluates the efficacy and safety of evinacumab, a novel LDLR-independent lipid-lowering therapy, in pediatric patients with homozygous familial hypercholesterolemia (HoFH).

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

17.

Refinement of the diagnostic approach for the identification of children and adolescents affected by familial hypercholesterolemia: Evidence from the LIPIGEN study.

Author: CasulaManuela, GazzottiMarta, CapraMaria Elena, OlmastroniElena, GalimbertiFederica, CatapanoAlberico L, PederivaCristina,

Effective FH diagnosis

This study describes the limitations of familiar hypercholesterolemia (FH) diagnosis in childhood, highlighting the need for additional information beyond typical features to improve diagnosis at a younger age.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

18.

Clinical Approach in the Management of Paediatric Patients with Familial Hypercholesterolemia: A National Survey Conducted by the LIPIGEN Paediatric Group.

Author: PederivaCristina, GazzottiMarta, ArcaMarcello, AvernaMaurizio, BanderaliGiuseppe, BiasucciGiacomo, BrambillaMarta, BuonuomoPaola Sabrina, CalabròPaolo, CipolloneFrancesco, CitroniNadia, D'AddatoSergio, Del BenMaria, GenovesiSimonetta, GuardamagnaOrnella, IannuzzoGabriella, IughettiLorenzo, MandraffinoGiuseppe, MaroniLorenzo, MombelliGiuliana, MuntoniSandro, NascimbeniFabio, PassaroAngelina, PellegattaFabio, PirroMatteo, PisciottaLivia, PujiaRoberta, SarzaniRiccardo, ScicaliRoberto, SuppressaPatrizia, ZambonSabina, ZentiMaria Grazia, CalandraSebastiano, CatapanoAlberico Luigi, TarugiPatrizia, GalimbertiFederica, CasulaManuela, CapraMaria Elena

FH treatment
Lipid-lowering

Early detection and treatment of familial hypercholesterolemia (FH) in children is crucial. This survey investigates the current clinical approach in managing pediatric patients with suspected FH in Italy. The findings highlight the need for standardized detection, management, and treatment of these patients.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

19.

Algorithms for Treating Dyslipidemia in Youth.

Author: KimGrace K, YeeJennifer K, BansalNidhi

Improved dyslipidemia management
Reduced CVD risk

Childhood initiation of statin therapy in youth under 18 years old with familial hypercholesterolemia reduces the risk of adult CVD. Current algorithms for dyslipidemia management in youth may be enhanced by incorporating recently approved pharmacotherapy and considering clinical risk scores for tailored treatment.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

20.

Dietary intervention for children and adolescents with familial hypercholesterolaemia.

Author: CapraMaria Elena, BiasucciGiacomo, CrivellaroElisa, BanderaliGiuseppe, PederivaCristina

Atherosclerosis prevention

Familial hypercholesterolaemia (FH) is a genetic disorder characterized by high cholesterol levels and premature atherosclerosis. This study reviews current dietary recommendations for FH in children and adolescents, highlighting the importance of individualised nutritional interventions to ensure adequate growth and development while managing cholesterol levels.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

21.

Prevalence of LDL-hypercholesterolemia and other cardiovascular risk factors in young people with type 1 diabetes.

Author: DrozdIrena, WeiskornJantje, LangeKarin, BiesterTorben, DatzNicolin, KapitzkeKerstin, ReschkeFelix, von dem BergeThekla, WeidemannJürgen, DanneThomas Paul Arthur, KordonouriOlga

LDL-hypercholesterolemia risk

This study investigated the prevalence of LDL-hypercholesterolemia and other cardiovascular risk factors in youth with Type 1 diabetes (T1D). 9% of youth with T1D had LDL-hypercholesterolemia, which was associated with increased blood pressure, insulin requirement, HbA1c, triglyceride, and total cholesterol, and a family history of premature CVD.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

22.

The impact of metabolic stressors on mitochondrial homeostasis in a renal epithelial cell model of methylmalonic aciduria.

Author: SchumannAnke, BrutscheMarion, HavermansMonique, GrünertSarah C, KölkerStefan, GroßOlaf, HannibalLuciana, SpiekerkoetterUte

Kidney disease
Metabolic stress

Methylmalonic aciduria (MMA-uria), a metabolic disorder caused by methylmalonyl-CoA mutase (MUT) deficiency, is associated with a high risk of chronic kidney disease (CKD). This study investigates the effects of metabolic stressors on renal mitochondrial energy metabolism in MMA-uria patients, providing insights into the potential mechanisms leading to CKD development.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

24.

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

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

Hypercholesterolemia treatment

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.

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.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
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.

Language : English

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