Effects of ivacaftor: A Synthesis of Findings from 24 Studies

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Original Abstract of the Article

Major Research Findings

Ivacaftor is a drug that has shown promise in treating cystic fibrosis (CF), potentially improving lung function, reducing inflammation, and preventing airway infections. Studies have shown that ivacaftor, when used in combination with other CFTR modulators, can improve the health of CF patients and their quality of life.

For instance, a study ( 10 ) revealed that children with CF who started lumacaftor/ivacaftor therapy during school age experienced improvements in paranasal sinus abnormalities. Another study ( 13 ) on children aged 2 to 5 years with CF who were homozygous for the F508del mutation, showed significant improvements in chest MRI scores when using lumacaftor/ivacaftor therapy, suggesting potential for early disease modification.

It is important to note that CFTR modulators, including ivacaftor, can vary in effectiveness from person to person, and not all patients experience a benefit. Side effects, such as mental health issues and liver abnormalities, have been reported.

Benefits and Risks

Benefit Summary

Ivacaftor has the potential to improve lung function in CF patients, reduce lung inflammation, and prevent airway infections. It may also enhance quality of life.

Risk Summary

Ivacaftor can cause side effects, including mental health issues and liver abnormalities. The effectiveness of ivacaftor can vary from person to person, and it may not be beneficial for all patients.

Comparison Across Studies

Commonalities

Multiple studies have indicated that CFTR modulators, including ivacaftor, can improve the overall health of CF patients and their quality of life. However, it is challenging to directly compare these studies because they used different drugs, dosages, and study designs.

Differences

The effectiveness of ivacaftor may vary depending on the study. For example, one study ( 10 ) showed improvements in paranasal sinus abnormalities in school-age children with CF, while another study ( 9 ) found no significant changes in airway inflammation in adult CF patients.

Consistency and Discrepancies

Multiple studies suggest that ivacaftor has the potential to improve the health of CF patients. However, not all patients benefit from it, and side effects can occur. Therefore, careful evaluation of the patient's condition is crucial before administering ivacaftor.

Practical Implications and Considerations

While ivacaftor holds promise as a treatment option for CF, its effectiveness can vary, and side effects are possible. Consequently, a thorough assessment of the patient's condition is essential before starting ivacaftor therapy.

Limitations of Current Research

More research on ivacaftor is needed, especially studies examining its long-term effects and safety. The precise mechanisms of action of ivacaftor remain to be fully elucidated.

Future Research Directions

Studies are needed to assess the long-term effects and safety of ivacaftor. Further research is also required to understand the mechanisms of action of ivacaftor and to develop more effective treatment options.

Conclusion

Ivacaftor has the potential to improve lung function in CF patients and enhance their quality of life. However, its effectiveness can vary, and side effects are possible. Careful evaluation of the patient's condition is essential before starting treatment with ivacaftor. Further research is expected to advance our understanding of ivacaftor and develop safer and more effective treatment options for CF.

Literature analysis of 24 papers
Positive Content
23
Neutral Content
0
Negative Content
0
Article Type
1
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1
24
2.

Effects of elexacaftor/tezacaftor/ivacaftor on liver fibrosis markers in adults with cystic fibrosis.

Author: TewkesburyDaniel H, ScottJennifer, BarryPeter J, Bright-ThomasRowland J, HanleyKaren Piper, AthwalVarinder, JonesAndrew M

Liver fibrosis improvement

Elexacaftor/tezacaftor/ivacaftor (E/T/I) showed no significant change in liver fibrosis markers except for APRI in CF adults. However, those with severe CF liver disease and an initial high liver stiffness measurement showed a significant reduction in LSM.

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

Language : English

4.

The Effects of Elexacaftor, Tezacaftor, and Ivacaftor (ETI) on Blood Glucose in Patients With Cystic Fibrosis: A Systematic Review.

Author: Salazar-BarraganMarcelo, TaubDaniel R

Improved glucose regulation

Cystic fibrosis (CF) is a genetic disorder caused by CFTR protein defects. This review examines the effects of a combination therapy of three CFTR modulators (elexacaftor, tezacaftor, and ivacaftor, or ETI) on blood glucose regulation in patients with the ΔF508 mutation.

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.

Effects of elexacaftor-tezacaftor-ivacaftor on daily treatment burden and airflow obstruction in adults with cystic fibrosis.

Author: TiotiuAngelica, IoanIulia, BillonYves

FEV1 improvement
Pulmonary exacerbation reduction
Respiratory symptom improvement

This study evaluated the impact of elexacaftor-tezacaftor-ivacaftor (ETI) on treatment burden and lung function parameters in patients with cystic fibrosis (CF) with at least one F508del mutation. The study aimed to assess the long-term real-world effects of ETI.

Observational StudyResearch that observes and collects data on participants' behavior and outcomes in their natural state, without intervention.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

6.

Effects of elexacaftor / tezacaftor / ivacaftor triple combination therapy on glycaemic control and body composition in patients with cystic fibrosis-related diabetes.

Author: GranciniValeria, GramegnaAndrea, ZazzeronLaura, AlicandroGianfranco, PorcaroLaura L, PiedepalumboFederica, LanfranchiChiara, DaccòValeria, OrsiEmanuela, BlasiFrancesco

Improved glucose metabolism
Increased fat mass
Reduced glucose variability

This study evaluated the effectiveness of elexacaftor/tezacaftor/ivacaftor (ETI), a triple combination therapy for cystic fibrosis (CF), on glucose metabolism, glucose variability, and body composition in CF patients with CF-related diabetes requiring insulin therapy. After six months of treatment, HbA1c and glucose variability decreased significantly, despite unchanged insulin requirements.

Observational StudyResearch that observes and collects data on participants' behavior and outcomes in their natural state, without intervention.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

7.

Longitudinal effects of elexacaftor/tezacaftor/ivacaftor on sputum viscoelastic properties, airway infection and inflammation in patients with cystic fibrosis.

Author: SchauppLaura, AddanteAnnalisa, VöllerMirjam, FentkerKerstin, KuppeAditi, BarduaMarkus, DuerrJulia, PiehlerLinus, RöhmelJobst, TheeStephanie, KirchnerMarieluise, ZiehmMatthias, LausterDaniel, HaagRainer, GradzielskiMichael, StahlMirjam, MertinsPhilipp, BoutinSébastien, GraeberSimon Y, MallMarcus A

Lung function improvement
Pulmonary exacerbation reduction

This study aimed to determine the longitudinal effects of elexacaftor/tezacaftor/ivacaftor (ETI) on airway mucus rheology, microbiome, and inflammation in cystic fibrosis (CF) patients with one or two F508del alleles throughout the first 12 months of therapy.

Observational StudyResearch that observes and collects data on participants' behavior and outcomes in their natural state, without intervention.
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

9.

ROCK STUDY in CF: sustained anti-inflammatory effects of lumacaftor-ivacaftor in sputum and peripheral blood samples of adult patients with cystic fibrosis-an observational study.

Author: AroojParniya, MorrissyDavid V, McCarthyYvonne, VaggTamara, McCarthyMairead, FlemingClaire, DalyMary, EustaceJoseph A, MurphyDesmond M, PlantB J

Cystic fibrosis treatment

This abstract discusses the potential effects of lumacaftor-ivacaftor (LUMA-IVA), a combination of CFTR corrector and potentiator, on proinflammatory cytokines (PICs) in patients with cystic fibrosis homozygous for the Phe508del CFTR mutation. It acknowledges that previous studies have shown clinical benefits of LUMA-IVA, but further research is needed to understand its impact on PICs.

Observational StudyResearch that observes and collects data on participants' behavior and outcomes in their natural state, without intervention.
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

10.

Long-term effects of lumacaftor/ivacaftor on paranasal sinus abnormalities in children with cystic fibrosis detected with magnetic resonance imaging.

Author: WucherpfennigLena, WuennemannFelix, EichingerMonika, SeitzAngelika, BaumannIngo, StahlMirjam, GraeberSimon Y, ZhaoShengkai, ChungJaehi, SchenkJens-Peter, AlrajabAbdulsattar, KauczorHans-Ulrich, MallMarcus A, SommerburgOlaf, WielpützMark O

CRS treatment
Lumacaftor/ivacaftor efficacy
Paranasal sinus improvement

This study evaluated the long-term effects of lumacaftor/ivacaftor treatment on paranasal sinus abnormalities in children with cystic fibrosis (CF). The study found that MRI detected improvements in paranasal sinus abnormalities in children who started treatment at school age and prevented an increase in abnormalities in children who started treatment at preschool age.

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

Language : English

13.

Effects of Lumacaftor/Ivacaftor on Cystic Fibrosis Disease Progression in Children 2 through 5 Years of Age Homozygous for <i>F508del-CFTR</i>: A Phase 2 Placebo-controlled Clinical Trial.

Author: StahlMirjam, RoehmelJobst, EichingerMonika, DoellingerFelix, NaehrlichLutz, KoppMatthias V, DittrichAnna-Maria, LeeChristopher, SommerburgOlaf, TianSimon, XuTu, WuPan, JoshiAniket, RayPartha, DuncanMargaret E, WielpützMark O, MallMarcus A

Disease modification
Growth parameters
Lung clearance index
Pancreatic function
Sweat chloride concentration

This Phase 2 study assessed the effects of LUM/IVA on CF disease progression in children 2 through 5 years of age with CF homozygous for F508del-CFTR. The study found that LUM/IVA led to improvements in chest MRI global score, LCI2.5, growth parameters, and biomarkers of pancreatic function. The safety profile was consistent with previous studies.

Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Clinical Trial, Phase IIA Phase II trial is a trial that further evaluates the efficacy and safety of a new drug or treatment, whose safety has been confirmed to some extent in a Phase I trial, in a small number of patients.
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

14.

Effects of Elexacaftor/Tezacaftor/Ivacaftor on Cardiorespiratory Polygraphy Parameters and Respiratory Muscle Strength in Cystic Fibrosis Patients with Severe Lung Disease.

Author: GiallongoAlessandro, ParisiGiuseppe Fabio, PapaleMaria, MantiSara, MuléEnza, AloisioDonatella, TerlizziVito, RotoloNovella, LeonardiSalvatore

Improved lung function
Quality of life improvement

This study investigates the effects of the triple therapy Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) on sleep-disordered breathing (SDB) and respiratory muscle strength in patients with cystic fibrosis (CF) and severe lung disease.

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

Language : English

16.

Effects of elexacaftor/tezacaftor/ivacaftor therapy in children with cystic fibrosis - a comprehensive assessment using lung clearance index, spirometry, and functional and structural lung MRI.

Author: StreibelCarmen, WillersCorin C, PusterlaOrso, BaumanGrzegorz, StranzingerEnno, BrabandtBen, BieriOliver, CurdyMarion, BulloMarina, FrauchigerBettina Sarah, KortenInsa, KrügerLinn, CasaultaCarmen, RatjenFelix, LatzinPhilipp, KieningerElisabeth

Milder CF course

This study investigates the effects of a combination regimen of elexacaftor, tezacaftor, and ivacaftor on lung abnormalities in patients with cystic fibrosis. The study aims to assess the treatment effect of this regimen on measures of structural and functional lung abnormalities using functional lung MRI with matrix-pencil decomposition.

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

18.

Real life evaluation of the multi-organ effects of Lumacaftor/Ivacaftor on F508del homozygous cystic fibrosis patients.

Author: Yaacoby-BianuKarin, SchnappZeev, KorenIlana, IlivitzkiAnat, KhatibMohamed, ShorbajiNadeem, ShteinbergMichal, LivnatGalit

Lung function improvement
Pulmonary exacerbation reduction

Lumacaftor/Ivacaftor (LUM-IVA) is a combination drug used to treat cystic fibrosis. While it has been shown to improve lung function, this study aimed to investigate the systemic effects of LUM-IVA on the body outside of the respiratory system.

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

21.

Effects of Elexacaftor/Tezacaftor/Ivacaftor Therapy on Lung Clearance Index and Magnetic Resonance Imaging in Patients with Cystic Fibrosis and One or Two <i>F508del</i> Alleles.

Author: GraeberSimon Y, RenzDiane M, StahlMirjam, PallenbergSophia T, SommerburgOlaf, NaehrlichLutz, BergesJulian, DohnaMartha, RingshausenFelix C, DoellingerFelix, VitzthumConstanze, RöhmelJobst, AllombaChristine, HämmerlingSusanne, BarthSandra, Rückes-NilgesClaudia, WielpützMark O, HansenGesine, Vogel-ClaussenJens, TümmlerBurkhard, MallMarcus A, DittrichAnna-Maria

Improved CFTR function
Improved lung ventilation
Reduced airway mucus plugging

Triple-combination CFTR modulator therapy with elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) improved lung ventilation and morphology in patients with cystic fibrosis (CF) and one or two F508del alleles. The study demonstrated improvements in lung clearance index (LCI) and magnetic resonance imaging (MRI) scores in these patients. The results suggest that ELX/TEZ/IVA can effectively improve lung function and address abnormalities in lung morphology in CF patients with F508del mutations.

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.
Observational StudyResearch that observes and collects data on participants' behavior and outcomes in their natural state, without intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

23.

Effects of ivacaftor on systemic inflammation and the plasma proteome in people with CF and G551D.

Author: HoppeJordana E, WagnerBrandie D, Kirk HarrisJ, RoweSteven M, HeltsheSonya L, DeBoerEmily M, SagelScott D

CFTR restoration

Ivacaftor is a drug that helps people with cystic fibrosis who have the G551D mutation. This study looked at how ivacaftor affects the body, particularly inflammation, growth, and certain proteins.

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

24.

Effects of Elexacaftor/Tezacaftor/Ivacaftor Therapy on CFTR Function in Patients with Cystic Fibrosis and One or Two <i>F508del</i> Alleles.

Author: GraeberSimon Y, VitzthumConstanze, PallenbergSophia T, NaehrlichLutz, StahlMirjam, RohrbachAlexander, DrescherMarika, MinsoRebecca, RingshausenFelix C, Rueckes-NilgesClaudia, KlajdaJan, BergesJulian, YuYin, ScheuermannHeike, HirtzStephanie, SommerburgOlaf, DittrichAnna-Maria, TümmlerBurkhard, MallMarcus A

Improved CFTR function
Improved clinical outcomes
Unknown

Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA), a combination of CFTR modulators, significantly improved CFTR function in both airway and intestinal epithelia in patients with cystic fibrosis and one or two F508del alleles. The study demonstrated the effectiveness of ELX/TEZ/IVA in restoring CFTR function and its potential for improving clinical outcomes.

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.
Observational StudyResearch that observes and collects data on participants' behavior and outcomes in their natural state, without intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

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