Pediatric pulmonology · 2026
Real-Life Experience With a Generic Formulation of Elexacaftor/Tezacaftor/Ivacaftor in Patients With Cystic Fibrosis With Responding Variants.
СтатьяНаблюдательное исследованиеНазвание и аннотация: Перевод пока не подготовлен
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Teper Alejandro, Rodríguez Viviana, Lubovich Silvina, Zaragoza Silvina, Rodríguez Novoa Ezequiel, García Bournissen Facundo
Аннотация
INTRODUCTION: Elexacaftor/Tezacaftor/Ivacaftor (ETI) has revolutionized cystic fibrosis (CF) care. In Argentina, the absence of patent restrictions allowed the production of a generic ETI formulation (ETIgf), significantly reducing costs.
OBJECTIVE: This study evaluates the real-world clinical impact of ETIgf in people with CF (pwCF).
METHODS: This prospective, single-center observational study in Buenos Aires, Argentina, included 71 pwCF (≥ 6 years) with responsive variants. Participants were categorized as: Group 1 (modulator-naïve) or Group 2 (previously on lumacaftor/ivacaftor). Clinical outcomes, including ppFEV1, LCI2.5, BMI z-score, CFQ-R, sweat chloride concentration (SCC), fecal elastase levels, liver enzymes and pulmonary exacerbations (PEx), were assessed over 12 months following the initiation of ETIgf.
RESULTS: Group 1 included 43 patients, and Group 2 included 28 patients (median age: 10.9 and 10.6 years, respectively). The mean percentage change from baseline after 12 months of treatment for Group 1 and Group 2 was: ppFEV1 +20.8 (p < 0.01) and +18.5 (p < 0.01); LCI2.5 -13.3 (p < 0.01) and -11.2 (p < 0.05); CFQ-R + 66.1 (p < 0.01) and +30.4 (p < 0.01); and SCC -53.1 (p < 0.01) and -58.2 (p < 0.01), respectively. BMI z-score increased by 0.5 in Group 1 (p < 0.001), while Group 2 showed a non-significant increase. Pulmonary exacerbations were reduced by 95% in both groups. No changes were observed in fecal elastase levels. Adverse events were mild: five patients experienced elevated transaminase levels and three presented with a rash.
CONCLUSION: ETIgf demonstrates robust clinical efficacy and safety, mirroring results reported for the originator. These findings highlight the viability of affordable generic modulators in expanding global access to life-changing therapy.
OBJECTIVE: This study evaluates the real-world clinical impact of ETIgf in people with CF (pwCF).
METHODS: This prospective, single-center observational study in Buenos Aires, Argentina, included 71 pwCF (≥ 6 years) with responsive variants. Participants were categorized as: Group 1 (modulator-naïve) or Group 2 (previously on lumacaftor/ivacaftor). Clinical outcomes, including ppFEV1, LCI2.5, BMI z-score, CFQ-R, sweat chloride concentration (SCC), fecal elastase levels, liver enzymes and pulmonary exacerbations (PEx), were assessed over 12 months following the initiation of ETIgf.
RESULTS: Group 1 included 43 patients, and Group 2 included 28 patients (median age: 10.9 and 10.6 years, respectively). The mean percentage change from baseline after 12 months of treatment for Group 1 and Group 2 was: ppFEV1 +20.8 (p < 0.01) and +18.5 (p < 0.01); LCI2.5 -13.3 (p < 0.01) and -11.2 (p < 0.05); CFQ-R + 66.1 (p < 0.01) and +30.4 (p < 0.01); and SCC -53.1 (p < 0.01) and -58.2 (p < 0.01), respectively. BMI z-score increased by 0.5 in Group 1 (p < 0.001), while Group 2 showed a non-significant increase. Pulmonary exacerbations were reduced by 95% in both groups. No changes were observed in fecal elastase levels. Adverse events were mild: five patients experienced elevated transaminase levels and three presented with a rash.
CONCLUSION: ETIgf demonstrates robust clinical efficacy and safety, mirroring results reported for the originator. These findings highlight the viability of affordable generic modulators in expanding global access to life-changing therapy.