Background: Acute pulmonary exacerbations are central events in the lives of individuals with cystic fibrosis (CF). Pulmonary exacerbations lead to impaired lung function, worse quality of life, and shorter survival. We hypothesized that aggressive early treatment of acute pulmonary exacerbation may improve clinical outcomes.
Purpose: Describe the rationale of an ongoing trial designed to determine the efficacy of home monitoring of both lung function measurements and symptoms for early detection and subsequent early treatment of acute CF pulmonary exacerbations.
Study design: A randomized, non-blinded, multi-center trial in 320 individuals with CF aged 14 years and older. The study compares usual care to a twice a week assessment of home spirometry and CF respiratory symptoms using an electronic device with data transmission to the research personnel to identify and trigger early treatment of CF pulmonary exacerbation. Participants will be enrolled in the study for 12 months. The primary endpoint is change in FEV1 (L) from baseline to 12 months determined by a linear mixed effects model incorporating all quarterly FEV1 measurements. Secondary endpoints include time to first acute protocol-defined pulmonary exacerbation, number of acute pulmonary exacerbations, number of hospitalization days for acute pulmonary exacerbation, time from the end of acute pulmonary exacerbation to onset of subsequent pulmonary exacerbation, change in health related quality of life, change in treatment burden, change in CF respiratory symptoms, and adherence to the study protocol.
Conclusions: This study is a first step in establishing alternative approaches to the care of CF pulmonary exacerbations. We hypothesize that early treatment of pulmonary exacerbations has the potential to slow lung function decline, reduce respiratory symptoms and improve the quality of life for individuals with CF.
Keywords: ABPA; AE; BCDM; Biostatistics and Clinical Data Management; CF; CFQ-R; CFRSD; Cystic Fibrosis Questionnaire-Revised; Cystic Fibrosis Respiratory Symptom Diary; Cystic fibrosis; DSMB; Data Safety Monitoring Board; Exacerbation; FEV(1); FVC; HADS; HRQOL; Health related quality of life; Hospital Anxiety and Depression Scale; MOS-SSS; Medical Outcomes Study Social Support Survey; PI; Principal Investigator; SAE; Spirometry; TAQ-CF; TDNCC; Therapeutics Development Network Coordinating Center; Treatment Adherence Questionnaire-CF; adverse event; allergic bronchopulmonary aspergillosis; cystic fibrosis; forced expiratory volume in one second; forced vital capacity; serious adverse event.