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

Pancreatic, nutritional and clinical outcomes in children 0-5 years with cystic fibrosis during the first 2 years of CFTR modulator therapy (PaNC): a multicentre prospective observational study protocol. (PubMed, BMJ Open) - P | "Infants and children 4 months to 5 years of age who are eligible for elexacaftor/tezacaftor/ivacaftor (ETI) or ivacaftor (IVA) meet the inclusion criteria for PaNC, with a total eligible cohort of 303 children at the commencement of recruitment...Additionally, a summary of non-identifiable results will be provided to CF consumers and CF healthcare providers via scientific and lay conferences and via peer-reviewed journals. ACTRN12624001185550; Pre-results."

Clinical data • Clinical protocol • Journal • Observational data • Cystic Fibrosis • Genetic Disorders • Immunology • Pediatrics • Pulmonary Disease • Respiratory Diseases • CFTR • ELANE

Real -World impact of previous exposure to CFTR modulators on clinical parameters and chronic sinus disease in people with cystic fibrosis on elexacaftor-tezacaftor-ivacaftor. (PubMed, Respir Med) - "ETI therapy was associated with improved clinical outcomes and improvement in sinus CT scans in pwCF and changes were independent of previous CFTR modulator therapies."

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

Condition-dependent effects of Elexacaftor/Tezacaftor/Ivacaftor (Trikafta) on Aspergillus fumigatus growth. (PubMed, Microbiol Spectr) - "By contrast, during macrophage infection, we observed that high concentrations of ETI treatment promoted fungal growth but inhibited inflammasome activation.IMPORTANCEThe advent of ETI therapy represents a pivotal moment, signaling the onset of major changes in the medical field of cystic fibrosis and its related infectious diseases. However, the impact of ETI treatment on the patient's microbiota and pathogens has to be further studied as proof arises of changes in patient colonization."

Journal • Allergic Bronchopulmonary Aspergillosis • Cystic Fibrosis • Genetic Disorders • Immunology • Infectious Disease • Pulmonary Disease • Respiratory Diseases

Development and Validation of a New LC-MS/MS Method for Simultaneous Quantification of Ivacaftor, Tezacaftor and Elexacaftor Plasma Levels in Pediatric Cystic Fibrosis Patients. (PubMed, Pharmaceuticals (Basel)) - " This method could be adopted to contemporarily measure ELX/TEZ/IVA plasma levels for both PK studies and monitor therapy compliance, especially in case of poor or partial responses to treatment, or to evaluate drug-drug interactions when multiple concomitant medications are required. Considering also the high cost burden of these medications to the health system, a TDM-based approach could facilitate more cost-effective patient management."

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

Blood platelet reduction after elexacaftor/tezacaftor/ivacaftor treatment in people with cystic fibrosis may depend on systemic inflammation reduction. (PubMed, Sci Rep) - "The reduction in platelets was significantly correlated with a decrease in leukocytes (rs: 0.352, p < 0.001), serum CRP levels (rs: 0.392, p < 0.001) and exacerbations (oral antibiotic cycles, rs: 0.241, p = 0.002; intravenous antibiotic cycles, rs: 0.153, p = 0.049). These findings suggest that the normalization of platelets may be dependent on the reduction in systemic inflammation induced by ETI therapy."

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

Respiratory Muscle Function in Children and Adolescents with Cystic Fibrosis in the Era of CFTR Modulator Therapies. (PubMed, Children (Basel)) - "Objective: The objective of this study was to analyze respiratory muscle function in children and adolescents with cystic fibrosis (CF) treated with Elexacaftor/Tezacaftor/Ivacaftor (ETI) compared to healthy individuals, based on the hypothesis that CFTR modulators may improve respiratory muscle strength... Children and adolescents with CF treated with ETI showed respiratory muscle strength comparable to that of healthy controls. Despite differences in lifestyle factors, these findings may reflect a positive impact of CFTR modulators on respiratory muscle function, although further longitudinal and controlled studies are needed."

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

Hemoglobin Levels in Children Treated for Cystic Fibrosis with CFTR Modulators: A Single Center Retrospective Study. (PubMed, J Clin Med) - "Background: An increase in hemoglobin (Hb) has been reported in subjects with CF treated with the CFTR modulator Ivacaftor and with the combination Lumacaftor/Ivacaftor (LI), while the literature about the impact of Elexacaftor/Tezacaftor/Ivacaftor (ETI) on Hb levels in the pediatric population is lacking. While the Hb response in those treated with ETI showed a transient reduction that lasted for one month, this may have depended on hemolysis, and returned to pre-treatment levels. Further studies will clarify the mechanisms that govern changes in Hb in subjects with CF treated with ETI."

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

Pulmonary Function Modulates Epigenetic Age in Subjects with Cystic Fibrosis. (PubMed, Int J Mol Sci) - "In the present study, we studied epigenetic age, applying the Horvath clock model, in 52 adult subjects with CF, all treated with elexacaftor/tezacaftor/ivacaftor (ETI)...This also translates into an increase in the physical activities of a group of subjects who, before the therapy, had grown up under a glass bell. The analysis of epigenetic age may represent a potential biomarker to assess the individual outcome of the therapy in subjects with CF, although long-term studies need to evaluate it."

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

"All my food is customized*": Barriers & facilitators concerning nutrition for persons living with cystic fibrosis. (PubMed, J Cyst Fibros) - "Understanding these supports and challenges is essential for developing effective and personalized nutritional strategies for PWCF. Enhancing access to knowledgeable dietitians, simplifying nutritional recommendations, and addressing cost barriers can significantly improve nutritional outcomes. Future research should focus on applicable solutions that utilize existing successful strategies while addressing common barriers many with CF face."

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

Elevated serum lipase in infants with cystic fibrosis exposed to prenatal and postnatal elexacaftor/tezacaftor/ivacaftor. (PubMed, J Cyst Fibros) - "These cases illustrate the relationship between sweat chloride as a biomarker of CFTR function, fecal elastase as a biomarker of pancreatic function, and serum lipase a biomarker of pancreatic cell injury. While prenatal ETI exposure can preserve pancreatic function in infants with CF, ongoing postnatal ETI exposure may be necessary to prevent pancreatic damage, suggesting a role for early ETI therapy in prenatally exposed infants."

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

N1303K (p.Asn1303Lys) Variant: Expanding Frontiers in the Treatment of Cystic Fibrosis. (PubMed, Respir Med) - "Faced with rapid clinical decline and ineligibility for approved therapies, a forskolin-induced swelling assay (FIS) on her intestinal organoids demonstrated a significant response to the triple CFTR modulator therapy Elexacaftor/Tezacaftor/Ivacaftor (ETI)...These infections were managed with personalized antimicrobial strategies, including inhaled meropenem and inhaled amphotericin, alongside ETI, leading to sustained clinical stability during an 8-month follow-up. This case highlights the potential of organoid-based testing to guide personalized CFTR modulator therapy for rare variants and underscores the importance of individualized antimicrobial strategies in addressing the complexities of CF. Expanding regulatory approval for modulators like ETI to include rare variants is essential for equitable CF care."

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

Adherence and perspectives of CF patients and caregivers on airway clearance therapies before and after elexacaftor/tezacaftor/ivacaftor (NACFC 2025) - No abstract available

Adherence • Clinical

Fecal microbiota changes in people with cystic fibrosis after 6 months of elexacaftor/tezacaftor/ivacaftor: Findings from the PROMISE study (NACFC 2025) - No abstract available

Cystic Fibrosis • Genetic Disorders • Immunology • Respiratory Diseases

Comparative clinical outcomes of Trikafta® versus bioequivalent ETI formulations in the management of Cystic Fibrosis in adults: a real world experience (NACFC 2025) - No abstract available

Clinical • Clinical data • Real-world • Real-world evidence • Cystic Fibrosis • Genetic Disorders • Immunology • Respiratory Diseases

Pregnancy and early parenthood does not have a detrimental impact on CF-health status in women under a dedicated CF Maternal Health Service in the elexacaftor/tezacaftor/ivacaftor era (NACFC 2025) - No abstract available

Clinical

Associations among body image dissatisfaction, body esteem, mental health, and elexacaftor/tezacaftor/ ivacaftor (ETI) adherence in people with cystic fibrosis. (NACFC 2025) - No abstract available

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

Modeling a single variable-time measurement of Elexacaftor/Tezacaftor/Ivacaftor in adults (NACFC 2025) - No abstract available

Clinical

Evaluating the clinical utility of oropharyngeal swabs from children with cystic fibrosis on elexacaftor/tezacaftor/ivacaftor using shotgun metagenomics (NACFC 2025) - No abstract available

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

An evaluation of fecal metabolomics and markers of intestinal integrity in individuals with advanced cystic fibrosis liver disease (aCFLD) and the effect of Elexacaftor-Tezacaftor-Ivacaftor (ETI) (NACFC 2025) - No abstract available

Clinical • Metastases • Cystic Fibrosis • Genetic Disorders • Hepatology • Immunology • Liver Cirrhosis • Respiratory Diseases

PGR--Physicians Grand Rounds (NACFC 2025) - "Know how to evaluate recurrent pancreatitis in a person with CF and the potential role of elexacaftor/tezacaftor/ivacaftor Describe and differentiate the manifestations of liver injury due to CF or elexacaftor/tezacaftor/ivacaftor. Learning Objectives: Describe the mechanism of different biologics and their role in the treatment of allergy bronchopulmonary aspergillosis in CF.Know how to evaluate recurrent pancreatitis in a person with CF and the potential role of elexacaftor/tezacaftor/ivacaftorDescribe and differentiate the manifestations of liver injury due to CF or elexacaftor/tezacaftor/ivacaftor."

Grand rounds • Hepatology • Immunology • Liver Failure • Pancreatitis • Pediatrics • Respiratory Diseases

Baseline lean mass deficits in adolescents and adults with CF persist 24-30m following elexacaftor/tezacaftor/ivacaftor initiation (NACFC 2025) - No abstract available

Clinical

Physiologically based pharmacokinetic of elexacaftor/tezacaftor/ivacaftor in children with cystic fibrosis: extrapolation of the dosing regimen in children under 2 years of age (NACFC 2025) - No abstract available

Clinical • PK/PD data • Cystic Fibrosis • Genetic Disorders • Immunology • Respiratory Diseases

Evaluation of testosterone levels in males with Cystic Fibrosis before and after initiation of Elexacaftor/Tezacaftor/Ivacaftor: Data from the PROMISE study (NACFC 2025) - No abstract available

Cystic Fibrosis • Genetic Disorders • Immunology • Respiratory Diseases

W28--Nutritional Breakthroughs: A Workshop on Evidenced-based Nutrition (NACFC 2025) - "Evaluate the impact of Elexacaftor-Tezacaftor-Ivacaftor (ETI) on fecal microbiota composition, intestinal inflammation, and metabolic profiles in individuals with CF. Apply emerging research findings from cohort studies to inform clinical care and personalized GI management in CF.Â"

Cystic Fibrosis • Gastrointestinal Disorder • Genetic Disorders • Hepatology • Immunology • Inflammation • Inflammatory Bowel Disease • Respiratory Diseases

Risk factors for pulmonary exacerbation in people with cystic fibrosis taking elexacaftor/tezacaftor/ivacaftor who stop chronic daily therapies (NACFC 2025) - No abstract available

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