Background
The Standardized Treatment of Pulmonary Exacerbations (STOP) program has the intent of defining best practices in the treatment of pulmonary exacerbations (PEx) in patients with cystic fibrosis (CF). The objective of this analysis was to describe the clinical presentations of patients admitted for intravenous (IV) antibiotics and enrolled in a prospective observational PEx study as well as to understand physician treatment goals at the start of the intervention.
Methods
We enrolled adolescents and adults admitted to the hospital for a PEx treated with IV antibiotics. We recorded patient and PEx characteristics at the time of enrollment. We surveyed treating physicians on treatment goals as well as their willingness to enroll patients in various study designs. Additional demographic and clinical data were obtained from the CF Foundation Patient Registry.
Results
Of 220 patients enrolled, 56% were female, 19% were adolescents, and 71% were infected with P. aeruginosa. The mean (SD) FEV1 at enrollment was 51.1 (21.6) % predicted. Most patients (85%) experienced symptoms for ≥7 days before admission, 43% had received IV antibiotics within the previous 6 months, and 48% received oral and/or inhaled antibiotics prior to IV antibiotic initiation. Forty percent had ≥10% FEV1 decrease from their best value recorded in the previous 6 months, but for 20% of patients, their enrollment FEV1 was their best FEV1 recorded within the previous 6 months. Physicians reported that their primary treatment objectives were lung function recovery (53%) and improvement of symptoms (47%) of PEx. Most physicians stated they would enroll patients in studies involving 10-day (72%) or 14-day (87%), but not 7-day (29%), treatment regimens.
Conclusions
Based on the results of this study, prospective studies are feasible and physician willingness for interventional studies of PEx exists. Results of this observational study will help design future PEx trials.
NAC recipients maintained their lung function while placebo recipients declined (24 week FEV1 treatment effect=150 mL, p<0.02). However no effect on HNE activity and other selected biomarkers of neutrophilic inflammation were detected. Further studies on mechanism and clinical outcomes are warranted.
Results from randomized prospective superiority and non-inferiority studies employing mean CRISS and FEV change from treatment initiation should prove compelling to the community. They will need to be large, but appear feasible.
Lung transplantation for CF is associated with acceptable survival rates and can improve quality of life. Lung transplant should be offered to all patients with advanced CF lung disease if they meet currently accepted inclusion and exclusion criteria.
Summary
Background
Although bronchoalveolar lavage (BAL) via flexible bronchoscopy is an essential diagnostic tool, its technique is not standardized in children. Our objective was to compare two different aspiration techniques of BAL in children (continuous wall suction vs. handheld syringe suction) in regards to the percentage of fluid recovered and the odds of performing a technically-acceptable procedure (i.e., >40% of volume return).
Methods
We conducted a review of all pediatric flexible bronchoscopies with BAL conducted at our institution over a two-year period. To minimize the differences between groups at baseline and reduce the possibility of bias, we used one-to-one propensity score (PS) caliper matching with no replacement for statistical analyses.
Results
We identified 539 procedures that met pre-specified criteria. There were considerable covariate imbalances between procedures in the handheld syringe group (n=147) and those in the continuous wall group (n=392); however, these imbalances were substantially reduced after the PS matching. In the matched sample (n=236), children in the handheld syringe group had ~7% higher volume return (95%CI=3.4–11.0, P<0.001) from BAL and three-fold higher odds (95%CI=1.5–8.6, P=0.002) of performing a technically-acceptable procedure.
Conclusions
Our results suggest that handheld syringe suction offers a higher percentage of volume return from BAL and increases the odds of performing a technically-acceptable procedure in children when compared to continuous wall suction.
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