Rationale and design of a randomized trial of home electronic symptom and lung function monitoring to detect cystic fibrosis pulmonary exacerbations: The early intervention in cystic fibrosis exacerbation (eICE) trial

Abstract

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 age 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.

Strategies to optimize treatment adherence in adolescent patients with cystic fibrosis

“…Home spirometry has been studied and used for clinical monitoring and early detection of exacerbations in CF with some success 58,59. It has not been evaluated as a tool for improving adherence, but there is patient interest in this kind of intervention 57…”

Tackling the increasing complexity of CF care

“…Other forms of telemedicine have also been evaluated but efficacy data are lacking to support its use in CF . Currently, an ongoing multicenter study (the early Intervention in CF Exacerbation study [eICE Study]) is assessing the role of home spirometry and symptom monitoring to identify and manage pulmonary exacerbations in adolescents and adults with CF . We will soon have the results of the eICE study to help understand whether such home monitoring is effective and in which CF populations it may be beneficial.…”

Early detection of pulmonary exacerbations in children with cystic fibrosis by electronic home monitoring of symptoms and lung function

“…Nevertheless, Sarfaraz et al found that daily home monitoring contributed to the early detection of PEx, which subsequently may have lowered the need for IV antibiotics 4 . Lechtzin et al conducted a randomised non-blinded multicentre trial in 320 CF patients (from 14 years) comparing usual care to home monitoring of spirometry and respiratory symptoms using the AM 2+ home monitor twice a week during one year 16 . However, the results of this trial have not been published yet.…”