Side Effects of lomitapide: A Synthesis of Findings from 12 Studies

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Original Abstract of the Article

Main Research Findings

Lomitapide is a licensed treatment for patients with homozygous familial hypercholesterolemia (HoFH) in the USA, the EU, Canada, and Mexico. 12

Lomitapide has been shown to be effective in reducing hepatic LDL-C production. 2 11

Lomitapide has been shown to significantly reduce LDL-C levels in HoFH patients. 11 8

Lomitapide has been shown to be effective in combination with other lipid-lowering medications. 3

Reasons for Side Effects

Lomitapide is an inhibitor of microsomal triglyceride transfer protein (MTP). 7 MTP plays a crucial role in lipid metabolism in the liver and small intestine. Lomitapide is thought to inhibit hepatic LDL-C production by blocking the function of MTP. However, since MTP plays a crucial role in lipid metabolism, lomitapide may affect lipid metabolism and cause side effects. 7

Common Side Effects

Gastrointestinal

The most common side effects of lomitapide are gastrointestinal side effects. 11 8

Liver

Lomitapide can cause fat to accumulate in the liver (hepatic steatosis). 11 9 Hepatic steatosis can occur if lomitapide is taken for a long period of time. 9

Countermeasures for Side Effects

Gastrointestinal

It is important to be mindful of your diet when taking lomitapide. 9 A high-fat diet may worsen gastrointestinal side effects. 9 In addition, lomitapide may interact with other medications. 8 Therefore, it is important to consult your doctor before taking lomitapide. 8

Liver

To prevent hepatic steatosis, it is important to undergo regular liver function tests. 11 If abnormalities are found in liver function tests, you may need to stop taking lomitapide. 11

Comparison between Studies

Commonalities

In several studies, lomitapide has been shown to significantly reduce LDL-C levels in HoFH patients. 11 8 3

Differences

There are some differences in research findings regarding the side effects of lomitapide. 11 8 9 One study reported hepatic steatosis as a major side effect. 11 On the other hand, another study reported gastrointestinal side effects as a major side effect. 8 These differences may be due to research design or participant characteristics. 9

Points to Note for Real-Life Application

Lomitapide may be an effective treatment for HoFH patients, but it is important to understand the risk of side effects. 8 When taking lomitapide, it is important to consult your doctor and follow their instructions. 8 It is also important to undergo regular liver function tests. 11

Limitations of Current Research

There are still limited studies that have evaluated the long-term safety of lomitapide. 11 Further research is needed to investigate the long-term effects of lomitapide. 11

Future Research Directions

Further research is needed to evaluate the long-term safety and efficacy of lomitapide. 11 Research is also needed to optimize the dosage and administration of lomitapide. 9

Conclusion

Lomitapide is an effective treatment that can significantly reduce LDL-C levels in HoFH patients. 11 However, lomitapide can cause side effects. 11 When taking lomitapide, it is important to consult your doctor and follow their instructions. 8

Literature analysis of 12 papers
Positive Content
11
Neutral Content
1
Negative Content
0
Article Type
1
0
0
6
12
1.

Established and Emerging Lipid-Lowering Drugs for Primary and Secondary Cardiovascular Prevention.

Author: MichaeliDaniel Tobias, MichaeliJulia Caroline, AlbersSebastian, BochTobias, MichaeliThomas

Reduced cardiovascular events

This review discusses established and emerging lipid-lowering drugs, including their mechanism of action, clinical trials, side effects, and effects on blood lipids. The review highlights the need for new treatment modalities that target specific cellular pathways to reduce cardiovascular risk in patients with dyslipidemia.

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

Language : English

2.

Lomitapide for treatment of homozygous familial hypercholesterolemia: The Québec experience.

Author: AljenedilSumayah, AlothmanLatifah, BélangerAlexandre M, BrownLeslie, LahijanianZubin, BergeronJean, CouturePatrick, BaassAlexis, RuelIsabelle, BrissonDiane, KhouryEtienne, GaudetDaniel, GenestJacques

HoFH treatment

The study describes the experience with lomitapide, an inhibitor of microsomal triglyceride transfer protein that lowers hepatic LDL-C production, in 79 Canadian 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

3.

Homozygous familial hypercholesterolemia in a young woman with dual gene mutations of low-density lipoprotein receptor and proprotein convertase subtilisin/kexin type 9.

Author: SuppressaPatrizia, CarbonaraConcetta, ScialpiNatasha, CiavarellaAlessandro, SabbàCarlo

Cholesterol lowering

This case report describes the successful treatment of a patient with a rare combination of homozygous LDLR and heterozygous PCSK9 mutations. The patient refused lipoprotein apheresis, so lipid-lowering therapy with statins, ezetimibe, and evolocumab was initiated. However, the desired low-density lipoprotein cholesterol target was not achieved. Dose-escalated lomitapide therapy was added, which enabled the patient to achieve the desired LDL cholesterol levels.

Case ReportsIn the medical field, a report that describes in detail a unique case, an unusual medical condition, or the effect of a treatment. Provides new insights and possibilities for treatment through individual patient cases.
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

4.

Therapeutic Options for Homozygous Familial Hypercholesterolemia: The Role of Lomitapide.

Author: GiammancoAntonina, CefalùAngelo B, NotoDavide, AvernaMaurizio R

Treats homozygous familial hypercholesterolemia

Lomitapide is a new drug for Homozygous Familial Hypercholesterolemia (HoFH) that works by inhibiting Microsomal Triglyceride Transfer Protein (MTP), thus reducing the production of harmful lipoproteins in the liver and intestines.

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

5.

Efficacy of Lomitapide in the Treatment of Familial Homozygous Hypercholesterolemia: Results of a Real-World Clinical Experience in Italy.

Author: D'ErasmoLaura, CefalùAngelo Baldassare, NotoDavide, GiammancoAntonina, AvernaMaurizio, PintusPaolo, MeddePaolo, VignaGiovanni Battista, SirtoriCesare, CalabresiLaura, PavanelloChiara, BucciMarco, SabbàCarlo, SuppressaPatrizia, NataleFrancesco, CalabròPaolo, SampietroTiziana, BigazziFederico, SbranaFrancesco, BonomoKatia, SileoFulvio, ArcaMarcello

Hypercholesterolemia treatment

This study evaluated the benefits of lomitapide in patients with homozygous familial hypercholesterolaemia (HoFH) who were already receiving standard clinical care.

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

Language : English

6.

Efficacy and Safety of Lomitapide in Hypercholesterolemia.

Author: LiuXin, MenPeng, WangYuhui, ZhaiSuodi, ZhaoZhigang, LiuGeorge

Reduced LDL-C

Statins are widely used to lower cholesterol levels, but they do not always reach the target levels in all patients, especially those with homozygous familial hypercholesterolemia (HoFH). Lomitapide is a new drug approved for treating HoFH, acting as a microsomal triglyceride transport protein (MTP) inhibitor to reduce proatherogenic lipoprotein 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

7.

Contemporary aspects of the biology and therapeutic regulation of the microsomal triglyceride transfer protein.

Author: HooperAmanda J, BurnettJohn R, WattsGerald F

Dyslipidemia treatment
MTP inhibitor effectiveness
Gastrointestinal side effects
Hepatic steatosis

This review focuses on the microsomal triglyceride transfer protein (MTP), its role in lipoprotein assembly, and its implications in abetalipoproteinemia, a rare genetic disorder characterized by MTP deficiency. The review explores MTP inhibitors, including lomitapide, and their potential benefits and limitations in treating dyslipidemia and cardiovascular disease.

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.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

8.

Lomitapide for the management of homozygous familial hypercholesterolemia.

Author: deGomaEmil M

Cardiovascular disease prevention
LDL-C reduction
Drug interactions
Gastrointestinal side effects
Hepatic steatosis
Transaminase elevations

This article discusses the use of lomitapide, a novel medication for homozygous familial hypercholesterolemia (HoFH), emphasizing the need for vigilant monitoring of liver function and potential drug interactions.

Case ReportsIn the medical field, a report that describes in detail a unique case, an unusual medical condition, or the effect of a treatment. Provides new insights and possibilities for treatment through individual patient cases.
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

9.

Potential approaches to ameliorate hepatic fat accumulation seen with MTP inhibition.

Author: LinMinjie, ZhaoShuiping, ShenLi, XuDanyan

Dyslipidemia therapy
Hepatic fat accumulation
Liver toxicity
Transaminase elevations

Lomitapide, a microsomal triglyceride transfer protein (MTP) inhibitor, is approved for homozygous familial hypercholesterolemia but can cause liver-related side effects. This review examines the mechanisms underlying these side effects and explores potential therapies for preventing them.

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.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

10.

New pharmacologic treatments for familial hypercholesterolemia.

Author: McDonoughAnnette, MaturaLea Ann, CarrollDiane

Improved heart health
Lower LDL cholesterol
Atherosclerosis
Coronary heart disease

Lomitapide and mipomersen are new treatments for familial hypercholesterolemia, a genetic disorder causing high cholesterol levels.

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

Language : English

12.

Pharmacokinetics and Pharmacodynamics of Lomitapide in Japanese Subjects.

Author: TaubelJorg, SumerayMark, LorchUlrike, McLeanAndre

Hypercholesterolemia treatment

This study compared the pharmacokinetics (PK), pharmacodynamics, safety, and tolerability of lomitapide, a licensed treatment for patients with homozygous familial hypercholesterolaemia, between Japanese and Caucasian subjects with elevated low-density lipoprotein cholesterol (LDL-C).

Clinical Trial, Phase IThe first stage of clinical trials in humans. Evaluates the safety, tolerability, and pharmacokinetics of a new drug/treatment.
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

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