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Background: Highly effective modulators of the CFTR channel have been demonstrated to dramatically impact disease progression and outcome. However, real-world data indicates that the magnitude of the clinical benefit is not equal among all patients receiving the treatment. We aimed to assess the variability in treatment response (as defined by the 6-month change in sweat chloride concentration, forced expiratory volume in one second [ppFEV1], body mass index [BMI], and CF Questionnaire-Revised [CFQ-R] respiratory domain score) and identify potential predictors in a group of patients receiving Elexacaftor-Tezacaftor-Ivacaftor (ETI) triple combination therapy., Methods: This was a single-center, prospective cohort study enrolling adults with CF at a major center in Italy. We used linear regression models to identify a set of potential predictors (including CFTR genotype, sex, age, and baseline clinical characteristics) and estimate the variability in treatment response., Results: The study included 211 patients (median age: 29 years, range: 12-58). Median changes (10-90th percentile) from baseline were: - 56 mEq/L (-76; -27) for sweat chloride concentration, +14.5 points (2.5; 32.0) for ppFEV1, +0.33 standard deviation scores (-0.13; 1.05) for BMI and +17 points (0; 39) for the CFQ-R respiratory domain score. The selected predictors explained 23 % of the variability in sweat chloride concentration changes, 18 % of the variability in ppFEV1 changes, 39 % of the variability in BMI changes, and 65 % of the variability in CFQ-R changes., Conclusions: This study highlights a high level of heterogeneity in treatment response to ETI, which can only be partially explained by the baseline characteristics of the disease., Competing Interests: Declaration of competing interest AG, CL, VD and FB reported honoraria for lectures from Vertex; AG reported honoraria for lectures from Zambon, Foodar, Insmed; FB reported honoraria for lectures from Viatris, Zambon, Foodar, Chiesi, Insmed; AG and VD reported travel support from Neupharma; AG and FB reported travel support from Viatris., (Copyright © 2024. Published by Elsevier B.V.)

Elexacaftor/tezacaftor/ivacaftor (ETI) is associated with major improvements in respiratory outcomes of individuals with cystic fibrosis (CF) and at least one Phe508del mutation. Although ETI was well tolerated in registration studies, the attention on adverse events not previously described is very high in the post-marketing phase. In this case series we report the onset of systemic arterial hypertension in 4 individuals with CF within the first weeks of starting therapy. All patients needed cardiac evaluation and started chronic anti-hypertensive therapy. Until more data is available, this report could foster the attention of CF physicians towards careful monitoring of cardiovascular parameters in patients starting ETI., Competing Interests: Declaration of Competing Interest The authors have no specific funding to report for this work., (Copyright © 2022. Published by Elsevier B.V.)

The prevalence of anti-SARS-CoV-2 antibodies in people with cystic fibrosis (CF) is largely unknown. We carried out a cross-sectional study between March and June 2021 with the aim of estimating the seroprevalence of anti-SARS-CoV-2 antibodies in two CF centres in Northern Italy. Total serum anti-SARS-CoV-2 (spike) antibodies levels were measured and values ≥0.8 U/mL were considered positive. Among 434 patients aged >12 years, 64 patients had a positive result (14.7%, 95% CI: 11.5-18.4), 36 (56.3%) without experiencing any COVID-19-related symptoms. Three out of 49 transplanted patients tested positive with an odds ratio for a positive result among transplanted as compared to non-transplanted patients of 0.35 (95% CI: 0.07-1.14). No significant differences were observed between sexes, age groups, socioeconomic status and lung disease severity. In conclusion, SARS-CoV-2 has infected a relatively high proportion of our patients but in most cases the infection was asymptomatic., Competing Interests: Declaration of Competing Interest None., (Copyright © 2021. Published by Elsevier B.V.)

Background: Over the last decades aggressive interventions have been successful to improve nutritional outcomes in people with cystic fibrosis (CF). As a result, with improvement of life expectancy and new CFTR modulators, overweight and obesity are progressively becoming a source of concern for adult population and in developed countries., Methods: This was a multicenter, observational, cross-sectional study of 321 adults with CF at three large CF centers in Italy. Patients were divided into three groups according to BMI classes, overweight and obesity (OW) group including patients with BMI ≥25 kg/m 2 , normal weight (NW) group with BMI 18.6-24.9 kg/m 2 and underweight (UW) group with BMI ≤18.5 kg/m 2 ., Results: We demonstrated that prevalence of OW in adults with CF in Italy is 22%. OW status is independently associated with male sex (OR 3.520, P = 0.001), pancreatic sufficiency (OR 2.873, P = 0.014) and older age at diagnosis (1.015, P = 0.042). BMI correlated with ppFEV1 (r = 0.337; P<0.0001) with median ppFEV1 significantly higher in patients with OW than comparisons. We also reported preliminary data on unfavorable cardiovascular risk factors in a subgroup of patients, where median blood levels [IQR] of cholesterol and systemic hypertension [%] were significantly higher in the OW group than in the NW and UW., Conclusions: People with CF and OW is a relevant patient group that might deserve better definition and proper clinical management., Competing Interests: Declaration of competing of interest The authors declare no conflict of interest., (Copyright © 2021. Published by Elsevier B.V.)