A multi-centre, randomized, controlled trial on coaching and telemonitoring in patients with cystic fibrosis: conneCT CF

Abstract

Background: The extend of lung disease remains the most important prognostic factor for survival in patients with cystic fibrosis (CF), and lack of adherence is the main reason for treatment failure. Early detection of deterioration in lung function and optimising adherence are therefore crucial in CF care. We implement a randomized controlled trial to evaluate efficacy of telemonitoring of adherence, lung function, and health condition in combination with behavior change interventions using innovative digital technologies.

Methods: This is a multi-centre, randomized, controlled, non-blinded trial aiming to include 402 patients ≥ 12 years-of-age with CF. A standard-of-care arm is compared to an arm receiving objective, continuous monitoring of adherence to inhalation therapies, weekly home spirometry using electronic devices with data transmission to patients and caring physicians combined with video-conferencing, a self-management app and professional telephone coaching. The duration of the intervention phase is 18 months. The primary endpoint is time to the first protocol-defined pulmonary exacerbation. Secondary outcome measures include number of and time between pulmonary exacerbations, adherence to inhalation therapy, changes in forced expiratory volume in 1 s from baseline, number of hospital admissions, and changes in health-related quality of life. CF-associated medical treatment and care, and health care related costs will be assessed by explorative analysis in both arms.

Discussion: This study offers the opportunity to evaluate the effect of adherence interventions using telemedicine capable devices on adherence and lung health, possibly paving the way for implementation of telemedicine in routine care for patients with CF.

Trial registration: This study has been registered with the German Clinical Trials Register (Identifier: DRKS00024642, date of registration 01 Mar 2021, URL: https://www.drks.de/drks_web/navigate.do?navigationId=trial.HTML&TRIAL_ID=DRKS00024642 ).

Keywords: Adherence; Cystic fibrosis; Home spirometry; Telemedicine.

Fig. 1

Standard of care for patients with CF compared to the intervention group in connect CF. A standard-of-care arm is compared to an intervention arm using objective, continuous monitoring of adherence to inhalation therapies, weekly home spirometry using electronic devices with data transmission to patients and caring physicians combined with a self-management app, video-conferencing and professional telephone coaching. In standard-of-care, scheduled visits with the caring CF physician take place once quarterly

Fig. 2

Study phases and procedures. During the preparation phase, participants are 1:1 randomized (R) to the intervention group (IG) and control group (CG), study procedures are explained and the correct handling of the study devices is being trained. The IG obtains a telemedicine capable nebulizer (eFlow rapid + nebulizer system, PARI Pharma GmbH, Germany), a home spirometry (mySpiroSense, PARI GmbH, Germany) and a CF therapy management app (PARI Connect App, PARI Pharma GmbH, Germany). The CG also receives a telemedicine capable nebulizer (eTrack Controller with eFlow rapid nebulizer, PARI Pharma Ltd, Germany, and 2net Hub, Philips, North America). During the phase of assessment of adherence, adherence to inhalation therapy is calculated. Adherence data will be available to the patient, caring CF physicians and coach in the IG, but not in the CG. At the end of the assessment phase, the IG is further stratified according to baseline adherence (adherence < 50% group and adherence ≥ 50% group). During the intervention phase, continuous digital monitoring of adherence to inhalation therapies and of lung function is performed in the IG. Participants of the IG can make use of video-conferencing with their caring CF physician up to three times per quarter. Participants of the IG with an adherence < 50% during the assessment phase additionally receive telephone coaching. In the case where mean adherence drops below 50% for at least four weeks in the first six months of the intervention phase in patients with initially good adherence (≥ 50%), these participants will receive a crisis talk followed by telephone coaching. In the last six months of the intervention phase, only monitoring and optional video-conferencing take place.  = quarterly visit outpatient clinic,  = intensive telephone coaching, telephone coaching, crisis phone call, t₀ = inclusion into study, t₁ = start assessment of adherence, t₂ = start study intervention, t₃ = six months of study intervention, t₄ = 12 months of study intervention, t₅ = end of study intervention