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  • ||||||||||  Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex
    Trial completion date, Trial primary completion date:  Trikafta for Patients With Non-cystic Fibrosis Bronchiectasis (clinicaltrials.gov) -  Jul 8, 2024   
    P4,  N=30, Recruiting, 
    Trial completion date: Jun 2024 --> Jul 2025 | Trial primary completion date: Jun 2024 --> Jul 2025
  • ||||||||||  Kalydeco (ivacaftor) / Vertex
    Evaluating serum lipid changes after Ivacaftor initiation in the GOAL study (206 AB) -  Jul 4, 2024 - Abstract #NACFC2024NACFC_634;    
    We speculate that this may be due to dietary modifications made by patients in response to weight gain on ivacaftor (average BMI increase of 1.4 kg/m2 from baseline at 18 months) or other factors that over time diminished the initial change in TC. Continued rise in lipids was not observed after the 3-month time point.
  • ||||||||||  Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex
    Longitudinal changes in bone mineral density in adolescents and adults with cystic fibrosis on elexacaftor/tezacaftor/ivacaftor  (104 AB) -  Jul 4, 2024 - Abstract #NACFC2024NACFC_629;    
    Despite the well-recognized systemic benefits of ETI, aBMD decreased by about 0.2 standard deviations in adolescents over the 24-month follow-up period but remained stable in adults. The potential impacts of ETI when initiated at young ages on bone strength, fracture risk, and adolescent bone accretion remain undefined but will be important considerations in screening and management guidelines.
  • ||||||||||  Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex
    The impact of diesel exhaust particles on cystic fibrosis lung disease (Theater A (Exhibit & Poster Hall)) -  Jul 4, 2024 - Abstract #NACFC2024NACFC_591;    
    In CF mice, DEPs and TGF-? acted synergistically to provoke airway smooth muscle hypertrophy and airway hyperreactivity.
  • ||||||||||  Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex
    Bioengineered 3D CF lung airway model for drug testing (Theater A (Exhibit & Poster Hall)) -  Jul 4, 2024 - Abstract #NACFC2024NACFC_590;    
    Our findings demonstrate that this novel model not only enables fabrication of human lung airway-like structures in vitro that mimic in vivo physiology, but also facilitates qualitative and quantitative measurement of patient-specific MCC and determines pharmacological effects. Our results suggest that this model is a valuable tool for understanding mechanisms underlying impaired MCC and testing the efficacy of novel therapeutic strategies for treatment of respiratory diseases such as CF.
  • ||||||||||  Q-Force (quercetin) / Quercis Pharma, Temple University, Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex
    Targeting cellular senescence in the cystic fibrosis lung. (Theater A (Exhibit & Poster Hall)) -  Jul 4, 2024 - Abstract #NACFC2024NACFC_584;    
    Senolytic treatment can reduce senescence markers and restore mucociliary clearance, and infection with P. aeruginosa can further exacerbate senescence in CF. Therefore, this study proposes cell senescence as a pathomechanism in the CF lung and that senolytics are a potential novel therapy for all PwCF independent of their CFTR modulator eligibility.
  • ||||||||||  Kalydeco (ivacaftor) / Vertex
    Characterizing small airway epithelia in cystic fibrosis ferrets with CFTR G551D mutations by bulk RNA-sequencing (Theater A (Exhibit & Poster Hall)) -  Jul 4, 2024 - Abstract #NACFC2024NACFC_583;    
    DE gene analysis of diseased small airway cells shows upregulation of genes and pathways associated with bacterial infection and inflammation. This study suggests that cultured CF small airway cells preserve native disease signatures and may serve as a model for testing potential therapies targeting inflammation and infection.
  • ||||||||||  Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex
    Role of pharmacogenomics in lung transplant with CF and Elexacaftor/Tezacaftor/Ivacaftor therapy (109 AB) -  Jul 4, 2024 - Abstract #NACFC2024NACFC_581;    
    Simplified and accessible pharmacogenomic data would augment clinical decision making in accounting for the influence of drug response along with existing consideration of all nongenetic factors. We hope to expand this work to include most of our CF LTx patient registry and to study effects on full pulmonary function, symptom score with the Cystic Fibrosis Questionnaire-Revised, impact on drug dosing, infection rate, diabetes mellitus control, and chronic rejection in these individuals.
  • ||||||||||  Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex
    The changing transplant landscape in the era of elexacaftor/tezacaftor/ivacaftor: a word of caution (109 AB) -  Jul 4, 2024 - Abstract #NACFC2024NACFC_579;    
    A word of caution is needed when discussing post-transplant expectations with potential transplant candidates because the prior transplant experience may not apply to this evolving population. It will be important to monitor this population to ensure updated and realistic expectations, particularly regarding survival estimates.
  • ||||||||||  SRI-41315 / Southern Research, Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex, eragidomide (CC-90009) / BMS
    Pharmacological rescue of CFTR premature termination codon (PTC) variants (157 A-C) -  Jul 4, 2024 - Abstract #NACFC2024NACFC_552;    
    CC-90009, G418, and SMG1i synergized to restore substantial G27X, G542X, R1158X, and W1282X CFTR activity but had negligible effect on E92X and Q890X CFTR. PTC CFTR-homozygous primary cells and cell lines are valuable tools to develop genotype-targeted, clinically relevant PTC readthrough and NMD-inhibiting agents.
  • ||||||||||  lumacaftor (VX-809) / Vertex, exaluren (ELX-02) / Eloxx Pharma, eragidomide (CC-90009) / BMS
    Rescue of CFTR nonsense mutations is enhanced under inflammatory stimuli (157 A-C) -  Jul 4, 2024 - Abstract #NACFC2024NACFC_549;    
    PTC CFTR-homozygous primary cells and cell lines are valuable tools to develop genotype-targeted, clinically relevant PTC readthrough and NMD-inhibiting agents. Cells were also treated in the last 24 hours with ELX-02 (200
  • ||||||||||  Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex
    Microenvironmental effects of a non-antibiotic therapy for a chronic airway disease modulate the virulence of its principal pathogen (156 A-C) -  Jul 4, 2024 - Abstract #NACFC2024NACFC_546;    
    A revolutionary therapy, elexacaftor/tezacaftor/ivacaftor (ETI), has improved mucociliary clearance and lung function and decreased infection in PwCF... These findings indicate that ETI not only benefits PwCF through improvement in lung function [2, 3], but may also reduce the virulence of its principal pathogen and highlight how the forces of microbial ecology can modulate polymicrobial infections through the modifications of microenvironmental chemistry.
  • ||||||||||  Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex
    Impact of elexacaftor/tezacaftor/ivacaftor on fecal elastase-1 in children with cystic fibrosis (Theater A (Exhibit & Poster Hall)) -  Jul 4, 2024 - Abstract #NACFC2024NACFC_493;    
    Because ETI is approved for children aged 2 and older, we may see greater frequency of improved pancreatic function in this younger population. Further longitudinal prospective studies are needed to better characterize children who may achieve improvement in pancreatic status after treatment with ETI.
  • ||||||||||  Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex
    Cystic fibrosis mortality trends in countries where people cannot access Elexacaftor/Tezacaftor/Ivacaftor therapy (205 A-C) -  Jul 4, 2024 - Abstract #NACFC2024NACFC_479;    
    The rise in CF mortality in countries where PwCF cannot access ETI therapy is likely attributable to the greater awareness of the disease and greater diagnostic capacity in those countries. Given the high cost of therapy, there is a risk of widening disparities in CF outcomes in the upcoming years in the absence of targeted interventions to ensure access to this life-saving treatment in resource-limited countries.
  • ||||||||||  Mycobutin (rifabutin) / Pfizer, Lupin, Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex
    Pharmacokinetics of elexacaftor/tezacaftor/ivacaftor with concomitant rifabutin (157 A-C) -  Jul 4, 2024 - Abstract #NACFC2024NACFC_476;    
    The PK of ETI is altered with concomitant RFB; however, the interaction is not as significant when compared with published data with rifampin. These data indicate a clinical trial evaluating the efficacy and safety of ETI with concomitant rifabutin treatment is warranted.
  • ||||||||||  Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex
    Feasibility and reliability of the YMCA 3 minute step test in adults with cystic fibrosis (153 A-C) -  Jul 4, 2024 - Abstract #NACFC2024NACFC_472;    
    Lower extremity musculo-skeletal pain and inability to keep the set pace of the test may be a barrier for some patients. Given that peak HR, peak %MaxHR, and recovery HBC had excellent test-retest reliability, any of these parameters could be used clinically to detect changes over time using MDC95% values calculated in this study.
  • ||||||||||  Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex
    Motivations and Barriers to Exercise for an adult CF population: a 5-year update (153 A-C) -  Jul 4, 2024 - Abstract #NACFC2024NACFC_471;    
    Ninety percent of participants in 2023 were on HEMT, predominantly elexacaftor/tezacaftor/ivacaftor, with varying levels of adherence to airway clearance and other respiratory therapies... The survey findings suggest that, while modulator therapy may influence exercise ability, individual factors such as preferences for exercise routines, motivations, and barriers, continue to influence patients
  • ||||||||||  Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex
    Elexacaftor/Tezacaftor/Ivacaftor on Gastrointestinal pH and Motility in CF Adults: Results from PROMISE (104 AB) -  Jul 4, 2024 - Abstract #NACFC2024NACFC_467;    
    Further work is necessary to determine the impact of CFTR modulators on intestinal acid-base transporters and the intestinal microbiome. ETI may change the motility of the proximal colon, but does not appear to affect the distal colon, potentially explaining the lack of change in bowel movements previously reported.
  • ||||||||||  Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex
    The relationship of the CF metabolome to treatment response following CFTR rescue (104 AB) -  Jul 4, 2024 - Abstract #NACFC2024NACFC_465;    
    The CF metabolome is highly predictive of ETI-induced changes in FEV1, SwCl, BMI, and symptoms. Novel metabolic endpoints of treatment efficacy may be useful in the assessment of new therapies in the CF pipeline and in identifying treatment targets that may complement the effects of modulators.
  • ||||||||||  Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex
    Home Spirometry as a CF Clinical Trial Endpoint: Results of the OUTREACH Study (210 A-C) -  Jul 4, 2024 - Abstract #NACFC2024NACFC_463;    
    P
    Novel metabolic endpoints of treatment efficacy may be useful in the assessment of new therapies in the CF pipeline and in identifying treatment targets that may complement the effects of modulators. One hundred fifteen participants were enrolled at 19 U.S. sites (mean
  • ||||||||||  Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex
    Long-term effects of elexacaftor/tezacaftor/ivacaftor therapy on airway and systemic inflammation in CF (210 A-C) -  Jul 4, 2024 - Abstract #NACFC2024NACFC_462;    
    ETI had significant, sustained effects on several measures of airway and systemic inflammation through 2 years of therapy in a large, multicenter cohort of adolescents and adults with CF, and changes in inflammation were correlated with clinical improvements. These anti-inflammatory effects are another disease-modifying benefit of this transformative therapy.
  • ||||||||||  Kalydeco (ivacaftor) / Vertex
    Recovery of pancreatic functions in CFTR-G551D ferrets after ivacaftor (104 C) -  Jul 4, 2024 - Abstract #NACFC2024NACFC_436;    
    The resident acinar cells in damaged CF pancreas likely contribute to regeneration. These results suggest that early initiation of highly effective modulator therapies may optimize clinical outcomes for exocrine and endocrine function in CFTR modulator therapy.
  • ||||||||||  Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex
    Making New Connections: An ACT-informed Telehealth Group for Teens with CF (102 AB) -  Jul 4, 2024 - Abstract #NACFC2024NACFC_428;    
    We have demonstrated the feasibility of implementing a virtual ACT-informed group at our center, with strong engagement of our patients. Future directions include expansion of recruitment, assessment of outcome measures (anxiety, depression, FEV1), and qualitative interviews with group participants to explore the efficacy of an ACT-informed telehealth intervention for adolescents with CF.