WS07.1 Standardized treatment of pulmonary exacerbations (STOP) study: Treatment goals for pulmonary exacerbations

Abstract

Objectives Pulmonary exacerbations (PEx) are common in individuals with CF, but there are no standard treatment recommendations. STOP collects information to define the primary endpoint for studies of treatment of PEx, and to access the feasibility for future trials to use the CF Foundation Patient Registry (CFFPR) for data capture. Methods STOP is an observational pilot study using the CFFPR and projected to enroll over 200 patients (age >12 years) at 10 sites admitted to the hospital for PEx treatment. A survey of admitting physicians was performed to ascertain treatment goals and physician willingness to enroll the patient in hypothetical interventional trials. Results After 12 months, 204 patients (56% female) have entered the study, with a mean age of 27.2±9.2 years and 53% homozygous F508del. The primary objective of treatment was recovery of lung function (53%) and improvement of symptoms (45%). The mean lung function recovery goal (target FEV1 – admission FEV1) was 13.7% predicted (range: 0–65.7%). For CF centers with a treatment protocol, the average planned treatment duration was 13.9±1.6 days. A majority of physicians were willing to enroll their patients in a trial of differing durations [10 days (71%) and 14 days (87%)], specified antibiotics (86%), and other treatments (83%), but less so for treatment of 7 days (30%). Conclusion The primary goals for treatment of PEx are recovery of lung function, targeting almost 14% recovery, and symptom improvement. Most sites express willingness to enroll patients into interventional trials of treatments. Early experience suggests the CFFPR can be used successfully for clinical trials. Supported by CFFT.