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Randomized Controlled Trial
. 2013 Nov;36(2):460-9.
doi: 10.1016/j.cct.2013.09.004. Epub 2013 Sep 19.

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

N Lechtzin  1 , N West, S Allgood, E Wilhelm, U Khan, N Mayer-Hamblett, M L Aitken, B W Ramsey, M P Boyle, P J Mogayzel Jr, C H Goss
Affiliations
Randomized Controlled Trial

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

N Lechtzin et al. Contemp Clin Trials. 2013 Nov.

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 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.

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Figures

Figure 1
Figure 1
This is a graph of cumulative study enrollment up to March 2013. The bars represent the number of study sites approved, yellow is projected and blue is actual. The lines represent subject enrollment, green is projected and purple is actual.
Figure 2
Figure 2
Schematic showing an overview of the study design and study visits.
See this image and copyright information in PMC

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