Side Effects of ivacaftor: A Synthesis of Findings from 20 Studies

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Original Abstract of the Article

Main Research Findings

Ivacaftor is a CFTR modulator used to treat cystic fibrosis (CF). This medication has shown promise in improving lung function and reducing sweat chloride levels in patients with CF. However, research on its long-term side effects is limited. 4 reported a rare case of breast development in a 7-year-old girl with CF who was taking ivacaftor. This study also found that higher plasma concentrations of ivacaftor did not correlate with improved lung function or sweat chloride levels. This suggests that some patients may be able to benefit from lower doses of ivacaftor, leading to the potential for personalized dosing.

Another study, 8 , observed seven adult CF patients who developed biliary colic after starting treatment with a triple-combination CFTR modulator therapy (elexacaftor/tezacaftor/ivacaftor). Six of these patients required cholecystectomy. This suggests a possible link between CFTR modulator therapy and biliary disease.

Additionally, 16 found that the same triple-combination therapy (elexacaftor/tezacaftor/ivacaftor) improved depressive symptoms in adult CF patients with at least one F508del mutation. This finding suggests a potential positive impact on mental health, although more research is needed.

While rash is a common side effect associated with CFTR modulators, 1 reported a case series where 19 CF patients experienced new onset or worsening acne after starting elexacaftor/tezacaftor/ivacaftor.

A separate study, 7 , reported testicular pain in seven young adult male CF patients within two weeks of starting elexacaftor/tezacaftor/ivacaftor. This potential side effect was not observed in clinical trials, and the exact mechanism remains unclear.

Reasons for Side Effects

The specific mechanisms behind ivacaftor's side effects are not fully understood, but they are likely related to the medication's interaction with CFTR protein and its impact on various bodily functions.

Common Side Effects

Breast Development

Breast development has been reported as a rare, dose-dependent side effect of ivacaftor. This side effect was seen in a 7-year-old girl with CF, as documented in 4 .

Biliary Disease

Biliary colic and cholecystectomy have been reported in patients who received elexacaftor/tezacaftor/ivacaftor, which contains ivacaftor, as reported in 8 .

Mental Health Changes

Improved depressive symptoms have been reported in some adult CF patients after receiving elexacaftor/tezacaftor/ivacaftor, as documented in 16 . However, there are also reports of potential negative impacts on mental health, including anxiety and neurocognitive issues, as described in 13 .

Acne

Acne has been reported as a possible side effect of elexacaftor/tezacaftor/ivacaftor in a case series of 19 CF patients, as found in 1 .

Testicular Pain

Testicular pain has been reported in seven young adult male CF patients within two weeks of starting elexacaftor/tezacaftor/ivacaftor, as documented in 7 .

Countermeasures for Side Effects

Breast Development

If you experience breast development while taking ivacaftor, consult with your doctor. They can determine the cause and recommend appropriate treatment.

Biliary Disease

If you experience biliary colic while taking elexacaftor/tezacaftor/ivacaftor, consult with your doctor. They can diagnose and treat any underlying biliary issues.

Mental Health Changes

If you experience any changes in mood or mental well-being while taking elexacaftor/tezacaftor/ivacaftor, consult with your doctor. Open communication is crucial for managing these side effects.

Acne

If your acne worsens while taking elexacaftor/tezacaftor/ivacaftor, consult with your doctor or a dermatologist. They can recommend appropriate treatment options.

Testicular Pain

If you experience testicular pain while taking elexacaftor/tezacaftor/ivacaftor, consult with your doctor immediately. They can investigate the cause and provide necessary treatment.

Comparison Between Studies

Commonalities Between Studies

All these studies highlight the importance of ongoing research to better understand the side effects of CFTR modulators, particularly ivacaftor. The studies suggest that individual patients may respond differently to these medications, emphasizing the need for personalized treatment approaches.

Differences Between Studies

The studies vary in their specific findings, with some focusing on particular side effects, such as breast development, biliary disease, or mental health changes. The observed side effects also differ in their severity and frequency.

Cautions Regarding Applications in Real Life

While CFTR modulators, including ivacaftor, offer significant benefits for CF patients, it is crucial to be aware of potential side effects. Open communication with your doctor is essential. Discuss any concerns or side effects you experience, and together you can determine the best course of action.

Limitations of Current Research

The research on ivacaftor's side effects is still ongoing, and more information is needed, particularly regarding long-term effects. The studies also vary in size and scope, and some focus on specific patient populations.

Future Research Directions

Future research should focus on expanding our understanding of ivacaftor's long-term effects and potential variations in individual responses. Researchers should also investigate personalized dosing strategies and explore ways to minimize or manage side effects.

Conclusion

Ivacaftor is a promising treatment for CF, but it is essential to be aware of its potential side effects. Open communication with your doctor is key to managing any adverse effects and ensuring the best possible outcome. Continued research is critical to improve our understanding of ivacaftor's long-term safety and effectiveness, as well as to develop personalized treatment approaches for CF patients.

Literature analysis of 20 papers
Positive Content
19
Neutral Content
0
Negative Content
1
Article Type
0
0
0
7
20
1.

Drug-induced acne with elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis.

Author: HudsonBrionna N, JacobsHollyann R, PhilbrickAlexander, ZhouXiaolong A, SimonsenMichelle M, SafirsteinJulie A, RotoloShannon M

Improved lung function
Reduced sweat chloride
Acne

This case series describes 19 patients who reported new onset or worsening of acne after starting treatment with the cystic fibrosis transmembrane conductance regulator (CFTR) modulator elexacaftor/tezacaftor/ivacaftor (ELX-TEZ-IVA). The mechanism and frequency of this adverse effect are unknown.

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

Language : English

2.

Ivacaftor: a novel gene-based therapeutic approach for cystic fibrosis.

Author: CondrenMichelle E, BradshawMarquita D

Convenient treatment option
Cystic fibrosis treatment
Drug-drug interactions

Ivacaftor is a new drug that targets the underlying genetic defect in cystic fibrosis. This study provides an overview of ivacaftor, its benefits, and its limitations.

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

Language : English

3.

Corticosteroid use and increased CXCR2 levels on leukocytes are associated with lumacaftor/ivacaftor discontinuation in cystic fibrosis patients homozygous for the F508del CFTR mutation.

Author: PohlKerstin, NicholsDavid P, Taylor-CousarJennifer L, SaavedraMilene T, StrandMatthew J, NickJerry A, BratcherPreston E

CF treatment
Respiratory side effects

Lumacaftor/ivacaftor (luma/iva) is a new drug that helps to improve the function of a protein that is defective in people with cystic fibrosis (CF). It can improve lung function, but some patients stop taking it because of side effects.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, N.I.H., ExtramuralResearch funding provided by the NIH to external research institutions and researchers.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

4.

Breast development in a 7 year old girl with CF treated with ivacaftor: An indication for personalized dosing?

Author: JeyaratnamJoshena, van der MeerRenske, BerkersGitte, HeijermanHarry Gm, BeekmanJeffrey M, van der EntCornelis K

CF treatment
CFTR modulator efficacy
Ivacaftor side effects

CFTR modulators have revolutionized cystic fibrosis treatment, but long-term side effects are still under investigation. This report describes a 7-year-old girl with CF who developed breast development as a rare dose-dependent side effect of ivacaftor treatment. The study also found that higher plasma concentrations of ivacaftor did not correlate with clinical effect, suggesting that some patients may benefit from lower doses.

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

5.

Advances in personalized medicine - medicinal chemistry and pharmacology of vemurafenib and ivacaftor.

Author: PellowskaM, MerkD, Schubert-ZsilaveczM

Personalized medicine
Side effect reduction
Treatment efficacy

Vemurafenib and ivacaftor, FDA-approved drugs targeting mutated proteins, show promising results in clinical trials with relatively mild side effects.

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

6.

A safety evaluation of ivacaftor for the treatment of cystic fibrosis.

Author: McColleySusanna A

Improved nutrition
Improved pulmonary function
Improved quality of life

This article reviews the safety profile of ivacaftor, a drug used to treat cystic fibrosis (CF) mediated by specific CFTR gene mutations. Studies have shown improved pulmonary function, nutrition, and quality of life in ivacaftor-treated patients.

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.

Testicular pain following initiation of elexacaftor/tezacaftor/ivacaftor in males with cystic fibrosis.

Author: RotoloShannon M, DuehlmeyerStephanie, SlackSarah M, JacobsHollyann R, HeckmanBrian

CF treatment
Impact on male fertility
Testicular pain

Elexacaftor/tezacaftor/ivacaftor (Trikafta) may cause testicular pain in some male patients with cystic fibrosis. This side effect typically resolves within a week and further research is needed to understand its long-term impact on fertility.

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.

Language : English

8.

Biliary disease and cholecystectomy after initiation of elexacaftor/ivacaftor/tezacaftor in adults with cystic fibrosis.

Author: SafirsteinJulie, GrantJonathan J, ClausenEmily, SavantDeepika, DezubeRebecca, HongGina

Biliary colic
Biliary tract disease
Gallbladder abnormalities

This case series describes seven patients with cystic fibrosis who experienced biliary colic shortly after starting treatment with elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA), a combination medication that improves cystic fibrosis transmembrane regulator (CFTR) function. Six of these patients required cholecystectomy, suggesting a possible association between ELX/TEZ/IVA and biliary complications.

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.

Language : English

9.

A current review of the safety of cystic fibrosis transmembrane conductance regulator modulators.

Author: GavioliElizabeth Marie, GuardadoNerli, HaniffFarah, DeiabNouran, ViderEtty

Improved clinical outcomes
Increased lifespans
Adverse drug reactions

This abstract discusses the increasing risk of adverse drug reactions in cystic fibrosis (CF) patients due to polypharmacy and CFTR modulators, particularly as CF patients continue to live longer.

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

11.

The impact of germline mutations on targeted therapy.

Author: SmithSimon A, FrenchTim, HollingsworthSimon J

Targeted therapy

Targeted therapies have a growing prominence in patient management and drug development. This review examines the impact of germline mutations on targeted therapies. Germline mutations play a crucial role in understanding disease mechanisms, drug metabolism, and exposure, and can be used to predict drug response and identify patients at risk for adverse reactions.

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

14.

Positive and negative impacts of elexacaftor/tezacaftor/ivacaftor: Healthcare providers' observations across US centers.

Author: BathgateChristina J, MutherEmily, GeorgiopoulosAnna M, SmithBeth, TillmanLaura, GrazianoSonia, VerkleijMarieke, LomasPaula, QuittnerAlexandra

Improved CF management
Neuropsychiatric changes

This study investigates the positive and negative impacts of elexacaftor/tezacaftor/ivacaftor (ETI), a treatment for cystic fibrosis (CF) associated with significant clinical improvements. It focuses on the reported neuropsychiatric changes associated with ETI.

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

Language : English

15.

Plasma and cellular ivacaftor concentrations in patients with cystic fibrosis.

Author: GuimbellotJennifer S, RyanKevin J, AndersonJustin D, ParkerKennedy L, Victoria OdomLauren, RoweSteven M, AcostaEdward P

Cystic fibrosis treatment
Tailored dosing

Ivacaftor is a medication for cystic fibrosis that helps the CFTR protein work better. This study found that patients taking ivacaftor have varying levels of the drug in their bodies, and some may benefit from lower doses.

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.
Research Support, N.I.H., ExtramuralResearch funding provided by the NIH to external research institutions and researchers.

Language : English

17.

Newly Discovered Cutting-Edge Triple Combination Cystic Fibrosis Therapy: A Systematic Review.

Author: DawoodSarah N, RabihAhmad M, NiajAhmad, RamanAishwarya, UpretyManish, CaleroMaria Jose, VillanuevaMaria Resah B, JoshaghaniNarges, VillaNicole, BadlaOmar, GoitRaman, SaddikSamia E, MohammedLubna

CFTR gene modulation
Pulmonary function
Quality of life
Headache
Liver function
Rash

This systematic review assesses the safety and efficacy of a triple therapy combining elexacaftor-tezacaftor-ivacaftor (Trikafta) for cystic fibrosis (CF) patients with at least one Phe508del mutation in the CFTR gene. The review concludes that Trikafta has shown significant improvement in pulmonary function, reduced exacerbations, and enhanced quality of life for CF patients.

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

18.

Elexacaftor, tezacaftor and ivacaftor: a case of severe rash and approach to desensitisation.

Author: ChengAnn, BakerOlivia, HillUta

Improved pulmonary function
Reduced infective exacerbation
Rash

A young adult male cystic fibrosis patient presented with severe rash following induction of elexacaftor, tezacaftor and ivacaftor (ELX/TEZ/IVA) triple therapy. Medication cessation was needed due to fever and widespread rash. Desensitization to ELX/TEZ/IVA was successful with incremental dosing and has allowed the patient to experience the transformative benefits of ELX/TEZ/IVA including improved pulmonary function and reduced episodes of infective exacerbation.

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.

Language : English

19.

The safety of lumacaftor and ivacaftor for the treatment of cystic fibrosis.

Author: Talamo GuevaraMaria, McColleySusanna A

CF treatment
Improved function
CYP3A interactions
Hepatic effects
Lumacaftor-ivacaftor
Ocular effects

Lumacaftor-ivacaftor is a medication used to treat cystic fibrosis. This article reviews the safety of this therapy.

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

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