Side Effects of tezacaftor and ivacaftor: A Synthesis of Findings from 21 Studies

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Original Abstract of the Article

Major Research Findings

Elexacaftor/tezacaftor/ivacaftor, a triple combination CFTR modulator, has shown significant improvements in lung function, weight, pancreatic function, mental state, and nasal symptoms in patients with cystic fibrosis (CF). 19 、 18 、 8 It may also be beneficial in improving chronic lung allograft dysfunction after lung transplantation in CF patients. 19 However, this medication has been associated with side effects such as mental status changes, worsening acne, decreased renal function, rash, gastrointestinal symptoms, and testicular pain. 10 、 9 、 4 、 12 、 7 Dosage adjustments based on individual patient needs are recommended. 4 、 2

Reasons for Side Effects

While the exact reasons for the side effects of elexacaftor/tezacaftor/ivacaftor are not fully understood, it's believed that these side effects are related to the medication's ability to improve CFTR protein function and its impact on various organs and tissues throughout the body. For instance, mental status changes might be linked to the CFTR protein potentially influencing the brain. 4 、 2 Worsening acne could be associated with the medication's potential to increase sebum production. 10 Decreased renal function might stem from the drug's potential effects on kidney function. 19 Rash could be an allergic reaction to the medication. 9 Gastrointestinal symptoms could be a consequence of the medication impacting gastrointestinal function. 20 Testicular pain might be related to the medication's possible effects on the male reproductive system. 7

Common Side Effects

Mental Status Changes

Elexacaftor/tezacaftor/ivacaftor may cause mental status changes. 4 、 2 These changes can include anxiety, irritability, sleep disturbances, and mental slowness. 2 If you experience these symptoms, it is crucial to consult your doctor.

Worsening Acne

Elexacaftor/tezacaftor/ivacaftor may worsen acne. 10 If you notice a worsening of your acne, seeking advice from a dermatologist is recommended.

Decreased Renal Function

Elexacaftor/tezacaftor/ivacaftor may cause a decrease in renal function. 19 If your renal function declines, consulting your doctor is essential.

Rash

Elexacaftor/tezacaftor/ivacaftor may cause rash. 9 If you develop a rash, it's important to discuss this with your doctor.

Gastrointestinal Symptoms

Elexacaftor/tezacaftor/ivacaftor might cause gastrointestinal symptoms. 20 Reported symptoms include stomach pain, nausea, and diarrhea. 20 It's advised to consult your doctor if you experience these symptoms.

Testicular Pain

Elexacaftor/tezacaftor/ivacaftor may cause testicular pain. 7 If you experience testicular pain, consult your doctor.

Countermeasures for Side Effects

Mental Status Changes

If you experience mental status changes, it is recommended to consult your doctor. 4 、 2 Depending on the situation, measures such as reducing the dosage or using alternative medications may be considered. 4 、 2

Worsening Acne

If your acne worsens, consult a dermatologist. 10 Acne treatment medications may be prescribed if needed.

Decreased Renal Function

If your renal function decreases, consult your doctor. 19 Using alternative medications may be considered if necessary.

Rash

If you develop a rash, consult your doctor. 9 Antihistamines or steroids may be prescribed if needed.

Gastrointestinal Symptoms

If you experience gastrointestinal symptoms, consult your doctor. 20 Antacids or antiemetics may be prescribed if necessary.

Testicular Pain

If you experience testicular pain, consult your doctor. 7 Pain relievers may be prescribed if needed.

Comparison Between Studies

Commonalities

Several studies have demonstrated that elexacaftor/tezacaftor/ivacaftor effectively improves lung function and weight in CF patients. 19 、 18 、 8 、 20 Additionally, numerous studies have reported side effects such as mental status changes, worsening acne, and rash. 10 、 9 、 4 、 2

Differences

Variations exist in the types and prevalence of side effects across different studies. 10 、 9 、 4 、 2 Some studies indicate the potential for elexacaftor/tezacaftor/ivacaftor to enhance lung function in patients following lung transplantation. 19 It's crucial to carefully consider these research findings in their entirety to determine the most appropriate treatment approach for each individual patient.

Precautions for Real-Life Applications

Elexacaftor/tezacaftor/ivacaftor holds the potential to improve the quality of life for individuals with CF. 19 、 18 However, the possibility of side effects necessitates adhering to your doctor's instructions regarding medication use. 10 、 9 、 4 、 2 Promptly seeking medical advice is essential if you experience any concerning symptoms, such as changes in mental state or rash. 4 、 9

Limitations of Current Research

Research on elexacaftor/tezacaftor/ivacaftor is still in its early phases, and further investigations are required to understand its long-term effects and potential side effects. 18 Moreover, because these studies have focused on specific populations (e.g. age or gender), the findings may not be universally applicable to all CF patients. 8

Future Research Directions

Continued research is necessary to thoroughly assess the long-term effects and side effects of elexacaftor/tezacaftor/ivacaftor. 18 Additional studies exploring the drug's efficacy in diverse populations are crucial. 8 Furthermore, research examining interactions between elexacaftor/tezacaftor/ivacaftor and other medications is highly important. 16

Conclusion

Elexacaftor/tezacaftor/ivacaftor is a novel treatment that has the potential to significantly improve lung function and weight in CF patients. 19 、 18 However, it's crucial to be aware of the potential for side effects and to take the medication as prescribed by your doctor. 10 、 9 、 4 、 2 Further research is necessary to gain a more comprehensive understanding of the safety and efficacy of elexacaftor/tezacaftor/ivacaftor. 18 、 8

Literature analysis of 21 papers
Positive Content
20
Neutral Content
0
Negative Content
1
Article Type
1
0
0
3
19
1.

Once-Daily Oral Sarecycline 1.5 mg/kg/day Is Effective for Moderate to Severe Acne Vulgaris: Results from Two Identically Designed, Phase 3, Randomized, Double-Blind Clinical Trials.

Author: MooreAngela, GreenLawrence J, BruceSuzanne, SadickNeil, TschenEduardo, WerschlerPhilip, Cook-BoldenFran E, DhawanSunil S, ForshaDouglass, GoldMichael H, GuenthnerScott, KempersSteven E, KircikLeon H, ParishJennifer L, RendonMarta I, RichPhoebe, Stein-GoldLinda, TyringStephen K, WeissRobert A, NasirAdnan, SchmitzCarsten, BoodhooTerry I, KaoukhovAlexandre, BerkDavid R

Acne treatment
Side effects

Sarecycline, a narrow-spectrum tetracycline antibiotic, was safe, well tolerated, and effective in patients with moderate to severe acne.

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.

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

9.

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

10.

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

11.

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

12.

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

13.

Real world outcomes of CFTR modulator therapy in Australian adults and children.

Author: KuekStephanie, McCullaghAngela, PaulEldho, ArmstrongDavid

CF treatment
Lung function
Nutrition
Adverse effects

CFTR modulators showed varying effectiveness in real-world settings. IVA and ELX/TEZ/IVA showed improvements in lung function and BMI, while LUM/IVA had a higher rate of adverse effects.

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

14.

Innovative Strategy toward Mutant CFTR Rescue in Cystic Fibrosis: Design and Synthesis of Thiadiazole Inhibitors of the E3 Ligase RNF5.

Author: BrusaIrene, SondoElvira, PesceEmanuela, TomatiValeria, GioiaDario, FalchiFederico, BalboniBeatrice, Ortega MartínezJose Antonio, VeronesiMarina, RomeoElisa, MargaroliNatasha, RecanatiniMaurizio, GirottoStefania, PedemonteNicoletta, RobertiMarinella, CavalliAndrea

Improve F508del-CFTR rescue
Increase F508del-CFTR corrector activity
Increase autophagy

A novel series of 1,2,4-thiadiazolylidene analogues were designed and synthesized as inhibitors of the ubiquitin ligase RNF5. Compound 16 demonstrated improved F508del-CFTR corrector activity compared to the original inhibitor, providing a potential new strategy for the treatment of cystic fibrosis (CF).

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

16.

Patient perspectives on elexacaftor/tezacaftor/ivacaftor after lung transplant.

Author: YoungDave, BartlettLauren E, GuimbellotJennifer, MilinicTijana, BurdisNora, GillEliana R, LeaseErika D, GossChristopher H, KapnadakSiddhartha G, RamosKathleen J

Improved outcomes
Quality of life
Cost
Drug interactions
Side effects

This study surveyed lung transplant recipients with cystic fibrosis (CF) about their perspectives on elexacaftor/tezacaftor/ivacaftor (ETI), a CFTR modulator that improves CF outcomes. The majority of respondents were aware of ETI but had concerns about potential drug interactions and side effects.

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

Language : English

17.

Experience With Elexacaftor/Tezacaftor/Ivacaftor in Patients With Cystic Fibrosis and Advanced Disease.

Author: Carrasco HernándezLaura, Girón MorenoRosa Mª, Balaguer CartagenaMari Nieves, PeláezAdrián, SoleAmparo, Álvarez FernándezAntonio, Felipe MontielAlmudena, OlveiraCasilda, OlveiraGabriel, Gómez BonillaAinhoa, Gómez CrespoBeatriz, García ClementeMarta, Solís GarcíaMarta, Quaresma VázquezJoana, Blitz CastroEnrique, Rodríguez GonzálezJesús, Expósito MarreroAndrea, Diab-CáceresLaila, Ramos HernándezCristina, Zamarrón de LucasEster, Prados SanchezConcha, Blanco AparicioMarina, López NeyraAlejandro, Sanz SantiagoVerónica, Luna ParedesCarmen, Delgado PecellínIsabel, Asensio de la CruzÓscar, Quintana GallegoEsther

Improved lung function
Improved quality of life
Reduced exacerbations
Unknown

Elexacaftor/tezacaftor/ivacaftor (ETI) effectively improved lung function, nutritional parameters, and quality of life in patients with cystic fibrosis.

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.

Language : English

19.

Elexacaftor/Tezacaftor/Ivacaftor as a Bridge to Lung Retransplant in a Recipient With Cystic Fibrosis.

Author: FitzMauriceThomas Simon, NazarethDilip, IyerKapil, WalshawMartin, Al-AloulMohamed

Improved lung function
Worsened immunosuppressant control

This case report describes the use of elexacaftor/tezacaftor/ivacaftor, a triple-combination cystic fibrosis transmembrane conductance regulator modulator, as a bridge to potential lung retransplant in a patient with cystic fibrosis and chronic lung allograft dysfunction.

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.

Language : English

20.

Real-life efficacy and safety of elexacaftor/tezacaftor/ivacaftor on severe cystic fibrosis lung disease patients.

Author: KosRenate, NeerincxAnne H, FennDominic W, BrinkmanPaul, LubRianne, VonkSteffie E M, RoukemaJolt, ReijersMonique H, Terheggen-LagroSuzanne W J, AltenburgJosje, MajoorChristof J, BosLieuwe D, HaarmanEric G, Maitland-van der ZeeAnke H,

CF treatment
Lung function
ETI use
Mild side effects

This study investigated the safety and efficacy of elexacaftor/tezacaftor/ivacaftor in patients with severe cystic fibrosis lung disease in a real-life setting, finding improved lung function, BMI, and reduced exacerbations with only mild side effects.

Observational StudyResearch that observes and collects data on participants' behavior and outcomes in their natural state, without intervention.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

21.

Concomitant Use of Elexacaftor/Tezacaftor/Ivacaftor and Etanercept in a Cystic Fibrosis Patient with Juvenile Idiopathic Arthritis.

Author: OrsiniSara Immacolata, MarraniEdoardo, PagniniIlaria, TaccettiGiovanni, TerlizziVito, SimoniniGabriele

CFTR modulator
Joint pain relief
Side effects

This report describes the first paediatric case of a patient with cystic fibrosis, Basedow's disease, and juvenile idiopathic arthritis treated with elexacaftor/tezacaftor/ivacaftor and anti-tumor necrosis factor.

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.

Language : English

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