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Reducing treatment burden is a priority for people with cystic fibrosis, whose health has benefited from using new modulators that substantially increase CFTR protein function. The SIMPLIFY study aimed to assess the effects of discontinuing nebulised hypertonic saline or dornase alfa in individuals using the CFTR modulator elexacaftor plus tezacaftor plus ivacaftor (ETI).The SIMPLIFY study included two parallel, multicentre, open-label, randomised, controlled, non-inferiority trials at 80 participating clinics across the USA in the Cystic Fibrosis Therapeutics Development Network. We included individuals with cystic fibrosis aged 12-17 years with percent predicted FEVFrom Aug 25, 2020, to May 25, 2022, a total of 672 unique participants were screened for eligibility for one or both trials, resulting in 847 total random assignments across both trials with 594 unique participants. 370 participants were randomly assigned in the hypertonic saline trial and 477 in the dornase alfa trial. Participants across both trials had an average ppFEVIn individuals with cystic fibrosis on ETI with relatively well preserved pulmonary function, discontinuing daily hypertonic saline or dornase alfa for 6 weeks did not result in clinically meaningful differences in pulmonary function when compared with continuing treatment.

RATIONALE: The etiology of cystic fibrosis (CF) pulmonary exacerbations (PEx) is likely multifactorial with viral, bacterial, and non-infectious pathways contributing. OBJECTIVES: To determine whether viral infection status and CRP (C-reactive protein) can classify subphenotypes of PEx that differ in outcomes and biomarker profiles. METHODS: Patients were recruited at time of admission for a PEx. Nasal swabs and sputum samples were collected and processed using the respiratory panel of the FilmArray multiplex polymerase chain reaction (PCR). Serum and plasma biomarkers were measured. PEx were classified using serum CRP and viral PCR: “pauci-inflammatory” if CRP

To analyze the efficacy and safety of octreotide treatment of persistent chylothorax in pediatric patients following cardiothoracic surgery.Retrospective chart review of patients admitted to the cardiac intensive care unit of a tertiary care center over a 10-year period (1998-2008). Nineteen patients were identified who underwent treatment with octreotide for persistent chylothorax following cardiothoracic surgery. We analyzed data regarding age, sex, type of cardiac lesion and surgical procedure, postoperative day octreotide therapy was initiated, maximum drainage prior to and during octreotide therapy, dose range of octreotide, days to resolution, and complications.Twelve patients (63%) experienced resolution of their chylothorax during octreotide treatment. Fourteen patients (74%) demonstrated a decrease in the peak effusion drainage rate following initiation of octreotide therapy, though two of these patients ultimately required further surgical intervention to achieve resolution. Octreotide treatment was associated with unchanged or increased effusion drainage rate in four patients (21%). Patients that responded to octreotide had a lower mean maximal drainage prior to octreotide initiation. Two patients experienced side effects temporally associated with octreotide therapy (transient hypoglycemia and diarrhea). No serious side effects were identified.Octreotide treatment of chylothorax in combination with dietary modifications in pediatric patients following cardiothoracic surgery resulted in a reduction of peak effusion drainage and eventual resolution in the majority of cases with few and transient side effects.