Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
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Therapies that target the underlying defect in Cystic Fibrosis (CF) will likely impact the future characteristics of the CF population and healthcare utilization. The objectives of this study were to estimate the potential impact of elexacaftor/tezacaftor/ivacaftor on morbidity and mortality, and the impact of delayed access. ⋯ Delayed access to elexacaftor/tezacaftor/ivacaftor will have a negative impact on lung health and survival in the CF population.
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The Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene is expressed in the heart, but this is not known to cause myocardial dysfunction or abnormalities in the ECG in people with CF. CFTR modulators such as tezacaftor/ivacaftor improve lung function and overall health in people with CF. ⋯ Drug withdrawal and reintroduction confirmed that neither drug alone, but only the combination, caused this change. As tezacaftor/ivacaftor is also present in elexacaftor/tezacaftor/ivacaftor, care may be needed to exclude this delayed interaction with azithromycin.
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Hyperosmolar hyperglycaemic state (HHS) has not previously been reported in cystic fibrosis-related diabetes (CFRD). We report the case of a 15-year old boy with stable CFRD who developed acute HHS after treatment with glucocorticoids and itraconazole for presumed allergic broncho-pulmonary aspergillosis (ABPA). This case highlights the dangerous and preventable combination of high glucose intake, glucocorticoids and itraconazole inhibition of CYP3A4 (with resultant glucocorticoid accumulation) that can result in a state of life- threatening HHS in an adolescent with previously stable CFRD.
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In Belgium, COVID-19 epidemy began on February 4, 2020 with a peak on April 10, 2020. Patients with cystic fibrosis (CF) followed in the Cliniques universitaires Saint-Luc were rapidly isolated before the government lockdown. ⋯ Even if respiratory symptoms linked to CF are frequent and compatible with COVID-19, anti-SARS-CoV-2 IgG antibodies were detected only in 3 asymptomatic patients. This reassuring study concerning the risk of COVID-19 in patients with CF illustrates the difficulty to distinguish COVID-19 symptoms from respiratory exacerbations and the need of generalized molecular testing to make a precise diagnosis.
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Major historical advances in cystic fibrosis (CF) respiratory clinical care, including mechanical airway clearance and inhaled medications, have aimed to address the consequences of cystic fibrosis transmembrane conductance regulator (CFTR) dysfunction. In contrast, CFTR modulator therapies instead target the underlying protein defect that leads to CF lung disease. ⋯ These changes could contribute substantially to the clinical benefit associated with modulator therapies, as well as providing an important indicator of treatment efficacy and residual pathophysiology. Indeed, the widespread introduction of modulator therapies might require us to re-consider our models of CF airway microbiology.