Obstructive Lung Disease
Improvements in pancreatic function biomarkers, including increases in fecal elastase-1 and decreases in serum immunoreactive trypsinogen, were seen in patients treated with lumacaftor and ivacaftor.
Inhaled liposomal ciprofloxacin is associated with a longer median time to first pulmonary exacerbation vs placebo in patients with non-cystic fibrosis bronchiectasis and chronic P aeruginosa lung infection requiring antibiotic therapy.
Due to brain excitability and hypoxia from coughing, patients with cystic fibrosis have a propensity for cortical spreading depression and hemiplegic migraine.
Recent guidelines set forth by an interdisciplinary panel assembled by the American Thoracic Society sought to demystify primary ciliary dyskinesia and address 4 main diagnostic questions.
Associations were seen for both Alzheimer's disease-related dementia and cerebrovascular etiologies.
Mild bronchiectasis in smokers is associated with distal pulmonary vascular pruning.
In infants with cystic fibrosis, preventive inhalation with hypertonic saline during the first months of life is safe and well tolerated and results in clinical improvements, according to a new study.
Including invalid data decreases the validity of multiple breath washout measurements in clinical trials involving adults with bronchiectasis.
VX-445-tezacaftor-ivacaftor led to noticeable improvements in Phe508del CFTR protein function.
Adding VX-659 to tezacaftor and ivacaftor therapy improved lung function in patients with cystic fibrosis.
Cystic fibrosis transmembrane conductance regulator dysfunction is associated with worsened airway disease in patients with COPD.
The FDA has granted Orphan Drug Designation to a novel inhaled treatment that could potentially improve lung function in patients with cystic fibrosis.
Forskolin-induced swelling in intestinal organoids is a clinically relevant biomarker of disease severity in infants diagnosed with cystic fibrosis.
Evidence is lacking that demonstrates that monotherapy with correctors has a clinically important effect on individuals with cystic fibrosis who have 2 copies of the F508del mutation.
First-phase and glucose-potentiation of arginine-induced insulin secretion as assessed by acute C-peptide responses improved after the initiation of ivacaftor in children with cystic fibrosis.
Lung transplantation can extend survival in patients with cystic fibrosis who have compromised lung function and increasing exacerbations.
Lung function in patients with primary ciliary dyskinesia was reduced compared with reference values from patients with cystic fibrosis in both sexes and in all age groups.
The approval was supported by data from ARRIVAL (N=25), an ongoing Phase 3 open-label safety study in children with CF aged 12 to <24 months with 1 of 10 CFTR gene mutations (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H).
In adult-diagnosed cystic fibrosis, diabetes, lung function, and age at diagnosis may predict patient survival.
The approval was based on data from a 24-week, open-label, Phase 3 study in patients aged 2 to 5 years.
A higher prevalence of anaerobic bacteria in cystic fibrosis was associated with higher lung function, less use of antibiotics, increased BMI, pancreatic sufficiency, and no requirement for insulin.
Results of a pooled analysis of cross-sectional studies demonstrated an increase in cough, sputum production, wheezing, and dyspnea from marijuana use.
Compared with placebo, the risk for a pulmonary exacerbation was reduced by 44% in participants with cystic fibrosis who received azithromycin.
Bronchiectasis-associated genera included Aspergillus, Cryptococcus, Clavispora, Botrytis, and Alternaria.
Ivacaftor decreased the risk for death, transplantation, hospitalization, and pulmonary exacerbation in patients with cystic fibrosis.
Sputum viscoelastic properties were associated with lung function and disease status in patients with cystic fibrosis.
Proteostasis Therapeutics is developing the combination treatment which includes a novel transmembrane conductance regulator (CFTR) amplifier (PTI-428), a third generation corrector (PTI-801) and a potentiator (PTI-808).
Patients with cystic fibrosis who took azithromycin had a significantly lower risk for detection of new nontuberculous mycobacteria, methicillin-resistant Staphylococcus aureus, and Burkholderia cepacia complex.
CFTR modulator therapy recommended in certain patients with cystic fibrosis.
Effective cystic fibrosis transmembrane conductance regulator modulator therapy may be of benefit in subsets of patients with cystic fibrosis.