Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex - LARVOL DELTA

Relating Improvement in Gastrointestinal (CFAbd-Score©) and Sinonasal (SNOT) Symptoms During Therapy With Elexacaftor/Tezacaftor/Ivacaftor in CF Patients. (PubMed, Pediatr Pulmonol) - "The burden of symptoms captured with the CFAbd-Score and the SNOT-20-GAV significantly and markedly decreased after ETI initiation. The modest association between changes in both organ-specific symptom scores supports the previously reported individualized electrophysiological responses observed in nasal-potential and intestinal-current measurements."

Journal • Cystic Fibrosis • Gastroesophageal Reflux Disease • Gastrointestinal Disorder • Genetic Disorders • Immunology • Pain • Pulmonary Disease • Respiratory Diseases

Phosphatidylserine liposomes for Mycobacterium abscessus infections management in people with cystic fibrosis non-eligible for CFTR modulators. (PubMed, Front Immunol) - "Here, we evaluated the in vitro therapeutic potential of PS-L in macrophages from people with cystic fibrosis (pwCF), either under therapeutic regimen or not with CFTR modulator therapy Elexacaftor/Tezacaftor/Ivacaftor (ETI). Finally, PS-L combined with amikacin further enhanced intracellular bacterial clearance compared to single treatments. Altogether, these findings support PS-L as a promising host-directed therapy against Mab infection, particularly for pwCF who cannot benefit from ETI."

Journal • Cystic Fibrosis • Genetic Disorders • Immunology • Infectious Disease • Inflammation • Pulmonary Disease • Respiratory Diseases • IL10 • IL1B • TNFA

Variable Response to Elexacaftor/Tezacaftor/Ivacaftor in [L467F;F508del] Homozygous Patients: A Case Series. (PubMed, Pediatr Pulmonol) - No abstract available

Journal

Trikafta for Patients With Non-cystic Fibrosis Bronchiectasis (clinicaltrials.gov) - P4 | N=32 | Completed | Sponsor: Emory University | Recruiting ➔ Completed

Trial completion • Bronchiectasis • Genetic Disorders • Immunology • Non‐Cystic Fibrosis Bronchiectasis • Pulmonary Disease • Respiratory Diseases

Modulation of host inflammatory pathways by Pseudomonas aeruginosa extracellular vesicles in cystic fibrosis: impact of pulmonary exacerbation and elexacaftor-tezacaftor-ivacaftor treatment. (PubMed, Front Immunol) - "P. aeruginosa EVs induced HBE cell inflammation, which was reduced in the presence of ETI."

Journal • Cystic Fibrosis • Genetic Disorders • Immunology • Infectious Disease • Inflammation • Pulmonary Disease • Respiratory Diseases • TNFA

Prenatal initiation of elexacaftor/tezacaftor/ivacaftor via carrier mother prevents congenital bilateral absence of vas deferens in a male infant with cystic fibrosis. (PubMed, J Cyst Fibros) - "This is the first report of a male infant with CF in whom prenatal ETI via a heterozygous carrier mother, started in the second trimester and continued postnatally, was associated with preserved exocrine pancreatic function, absence of pulmonary disease, and presence of vas deferens. These findings suggest that prenatal CFTR modulation - even when initiated late in gestation - may alter the trajectory of CF-related organ manifestations, including male reproductive development. Long-term follow-up is essential to determine whether these early benefits translate into sustained preservation of fertility and multiorgan function."

Journal • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

"Side versus adverse effects of elexacaftor/tezacaftor/ivacaftor therapy in people with CF". (PubMed, Respir Med) - "ETI demonstrates robust real-world efficacy in adults with CF. Side effects due to CFTR restoration are generally self-limiting, whereas adverse effects from drug toxicity persist longer and are more frequently cause for permanent discontinuation of ETI."

Adverse events • Journal • Cough • Cystic Fibrosis • Fatigue • Genetic Disorders • Immunology • Pain • Pulmonary Disease • Respiratory Diseases

Changes in the fungal ecology in the era of CFTR modulators: Results from a French multicenter study focused on cystic fibrosis airways. (PubMed, Med Mycol) - "Data from pwCF followed at CF reference centers in Besançon, Bordeaux, Limoges, and Rennes were collected before CFTRmt use (2014) and after their widespread implementation (2022), including elexacaftor/tezacaftor/ivacaftor (ETI) as well as other CFTR modulator therapies used in France...CFTR modulators appear to modify the airway mycobiome and fungal ecology depending on CFTRmt type. Among several factors that may account for these mycobiome changes between 2014 and 2022, environmental changes, including climate-related shifts in Aspergillus distribution, may contribute potentially."

Clinical • Journal • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Detection of Pseudomonas aeruginosa in cystic fibrosis after initiation of CFTR modulators: a systematic review. (PubMed, Paediatr Respir Rev) - "The advent of elexacaftor/tezacaftor/ivacaftor (ETI) has transformed clinical outcomes, but its impact on airway microbiology and diagnostic surveillance remains uncertain...However, the clinical and inflammatory significance of PCR+/culture- results remains uncertain. Combining molecular and culture-based surveillance may help guide infection monitoring in the modulator era."

Journal • Review • Cystic Fibrosis • Genetic Disorders • Immunology • Infectious Disease • Pediatrics • Pulmonary Disease • Respiratory Diseases

Longitudinal changes in bone mineral density after initiation of elexacaftor-tezacaftor-ivacaftor in youth and adults with cystic fibrosis: PROMISE-ENDO. (PubMed, J Cyst Fibros) - "Youth aBMDZ was lower at multiple skeletal sites 12-18 mos after ETI initiation, and these changes persisted at 24-30 mos. Adult aBMDZ generally remained unchanged."

Journal • Cystic Fibrosis • Genetic Disorders • Immunology • Musculoskeletal Diseases • Orthopedics • Pulmonary Disease • Respiratory Diseases

Determinants of early Elexacaftor-Tezacaftor-Ivacaftor use in adults with cystic fibrosis and preserved lung function: insights from a European multicenter survey (IWLH 2026) - "Although several disease markers more consistently support the decision to initiate ETI in individuals with preserved lung function, the absence of clear clinical guidance contributes to heterogeneous prescribing practices across centres. This highlights the need for longitudinal studies to clarify the long-term outcomes of ETI in this population"

Clinical • Cystic Fibrosis • Genetic Disorders • Immunology • Respiratory Diseases

CFTR modulators partially restore the epithelial interferome in Aspergillus infection to improve clinical outcome. (PubMed, EBioMedicine) - "CFTR modulators have led to improved clinical outcomes in CF related Aspergillus-related lung disease potentially due to partial restoration of the host antifungal epithelial type I/III interferon response. Exogenous IFNλ1 further improved antifungal killing capacity of CF-neutrophils presenting a plausible future therapeutic strategy."

Clinical data • Journal • Cystic Fibrosis • Genetic Disorders • Immunology • Infectious Disease • Pulmonary Disease • Respiratory Diseases

Evaluation of the Need for Comprehensive Care for Patients with Cystic Fibrosis. (PubMed, J Am Board Fam Med) - "There is a gap in healthcare for pwCF, especially for those who do not routinely interact with a PCP. CF clinicians may not be up to date on primary care management and PCPs may not be comfortable with treating patients with a high acuity chronic condition. PCPs should be better integrated into the CF care team to ensure pwCF are receiving comprehensive care."

Journal • Cystic Fibrosis • Genetic Disorders • Immunology • Infectious Disease • Influenza • Oncology • Pneumococcal Infections • Preventive care • Pulmonary Disease • Respiratory Diseases • CFTR

CFTR Modulator Therapy and Glycemic Control: A Meta-Analysis. (PubMed, Pediatrics) - "ETI therapy significantly improves glycemic control in CF, particularly when initiated at a young age. Further research is needed to confirm and detail these findings."

Journal • Retrospective data • Cystic Fibrosis • Diabetes • Genetic Disorders • Immunology • Metabolic Disorders • Pulmonary Disease • Respiratory Diseases

Comparison of Stool Microbiome in Children with Cystic Fibrosis Treated with and Without Elexacaftor-Tezacaftor-Ivacaftor-A Pilot Study. (PubMed, Int J Mol Sci) - "Alpha diversity showed a negative trend with the duration of ETI therapy for both bacteriome and mycobiome in children with CF treated with ETI. Future studies are needed to confirm or refute our preliminary findings."

Journal • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Upregulation of a CFTR mRNA isoform has therapeutic potential for the treatment of 3' CFTR PTC variants. (PubMed, Mol Ther Nucleic Acids) - "We demonstrate that e22 trunc mRNA generates a truncated CFTR protein whose Cl- transport function can be enhanced with elexacaftor/tezacaftor/ivacaftor (ETI) treatment. ASO and ETI treatment in combination restore ∼20% and 25% of wild-type CFTR Cl- transport function in immortalized epithelial and primary hBE cells homozygous for CFTR W1282X, respectively. This study provides a foundation for advancing ASO-mediated upregulation of e22 trunc mRNA and protein as a therapeutic approach for cystic fibrosis caused by 3'-terminal CFTR PTC mutations."

Journal • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases • CFTR

How do I treat : allergic bronchopulmonary aspergillosis in patients with cystic fibrosis (PubMed, Rev Med Liege) - "Treatments include systemic corticosteroids, antifungals, and monoclonal antibodies such as omalizumab and mepolizumab. The triple combination of elexacaftor, tezacaftor, and ivacaftor indirectly improves ABPA by reducing inflammation and enhancing bronchial drainage. After presenting a complex clinical case, this article provides a synthesis of the diagnostic criteria for ABPA, and the different treatments."

Clinical guideline • Journal • Allergic Bronchopulmonary Aspergillosis • Cystic Fibrosis • Genetic Disorders • Immunology • Inflammation • Pulmonary Disease • Respiratory Diseases

Impact of elexacaftor/tezacaftor/ivacaftor therapy on microbiological parameters in respiratory samples from patients with cystic fibrosis (ESCMID Global 2026) - No abstract available

Clinical • Cystic Fibrosis • Genetic Disorders • Immunology • Respiratory Diseases

Comparison of Single-Breath and Multi-Breath Xe-MRI in the Longitudinal Assessment of Treatment in Children With Cystic Fibrosis. (PubMed, J Magn Reson Imaging) - "CoVFV may continue to evolve over 2 years in pediatric CF patients receiving ETI, particularly in individuals with persistent ventilation defects."

Journal • Cystic Fibrosis • Genetic Disorders • Immunology • Pediatrics • Pulmonary Disease • Respiratory Diseases

Impact of ETI on cholesterol loading and efflux capacity in a cohort of adult CF patients (IWLH 2026) - "The aim of this longitudinal study was to describe changes in HDL Cholesterol Efflux Capacity (CEC) and Cholesterol Loading Capacity (CLC) in awCF after starting therapy with Elexacaftor/Tezacaftor/Ivacaftor (ETI)... Concluding, our results suggest that the correction of CFTR improves HDL function, possibly exerting a protective cardiovascular effect. Larger population studies with longer follow-up time should certainly be conducted to validate these results and provide a clinical confirmation of atherosclerotic risk reduction induced by ETI in awCF."

Clinical • Atherosclerosis • Cardiovascular • ABCA1 • ABCG1

Effects of Elexacaftor Tezacaftor Ivacaftor on sinonasal imaging in children with cystic fibrosis. (PubMed, Rhinology) - "This study highlights ETI's efficacy in improving sinonasal involvement in children aged 6 to 12 with CF. This is in line with the observations of clinical improvement, and presents an alternative to sinus surgery, thus potentially leading to a reduction in surgical interventions."

Journal • Cystic Fibrosis • Genetic Disorders • Immunology • Otorhinolaryngology • Pulmonary Disease • Respiratory Diseases

Epithelial extracellular vesicles induce inflammation and neutrophil activation in the Pseudomonas aeruginosa infected cystic fibrosis bronchial epithelium. (PubMed, Front Immunol) - "In addition, the effects of EVs on neutrophil migration and activation were determined as well as the role of CFTR deficiency by using CFTR modulator therapy (Elexacaftor/Tezacaftor/Ivacaftor)...Interestingly, the effect of EVpPAs on inflammation was not attenuated by pre-treatment with ETI. EVs from the PA-infected CF bronchial epithelium seem to facilitate an autocrine and paracrine pro-inflammatory response that is not attenuated by ETI treatment, suggesting a novel contribution of EVs to the chronic inflammatory phenotype observed in the PA-infected CF lung."

Journal • Bronchiectasis • Cystic Fibrosis • Genetic Disorders • Immunology • Infectious Disease • Inflammation • Pulmonary Disease • Respiratory Diseases • CXCL8 • IL6 • TNFA

Evolution of the respiratory microbiological profile in people with cystic fibrosis undergoing treatment with elexacaftor/tezacaftor/ivacaftor (ESCMID Global 2026) - No abstract available

Cystic Fibrosis • Genetic Disorders • Immunology • Respiratory Diseases

Impact of Elexacaftor-Tezacaftor-Ivacaftor Treatment on Metabolic, Epigenetic and Fecal Microbiota Profiles in People With Cystic Fibrosis. (clinicaltrials.gov) - P=N/A | N=150 | Recruiting | Sponsor: Meyer Children's Hospital IRCCS

New trial • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Modulation of immune responses by elexacaftor/tezacaftor/ivacaftor therapy in cystic fibrosis: data from a compassionate use program. (PubMed, Respir Res) - "Long-term ETI treatment confirms clinical benefits and exerts measurable immunomodulatory effects, partially via inhibition of JAK/STAT signaling. These findings support its broader impact beyond CFTR correction. Further studies are warranted to explore long-term immunological outcomes, especially in younger patients initiating early therapy."

Journal • Cystic Fibrosis • Genetic Disorders • Immunology • Inflammation • Pulmonary Disease • Respiratory Diseases • CRP • CXCL8 • IL6 • STAT5 • TNFA