Optimizing CFTR modulator therapy management for cystic fibrosis through the ReX platform

BackgroundCystic fibrosis (CF) is a chronic multi-systemic disease that requires a complex daily treatment regimen. Therefore, there is sub-optimal adherence to CF therapies, and it was shown to impact its clinical and economic burden. Cystic fibrosis transmembrane conductance regulator modulators (...

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Main Authors: Karin Yaacoby-Bianu, Malena Cohen-Cymberknoh, David Shoseyov, Tal Lavi, Ana Ostrovski, Michal Shteinberg, Galit Livnat
Format: Article
Language:English
Published: Frontiers Media S.A. 2023-12-01
Series:Frontiers in Pediatrics
Subjects:
Online Access:https://www.frontiersin.org/articles/10.3389/fped.2023.1300968/full
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author Karin Yaacoby-Bianu
Karin Yaacoby-Bianu
Malena Cohen-Cymberknoh
David Shoseyov
Tal Lavi
Ana Ostrovski
Michal Shteinberg
Michal Shteinberg
Galit Livnat
Galit Livnat
author_facet Karin Yaacoby-Bianu
Karin Yaacoby-Bianu
Malena Cohen-Cymberknoh
David Shoseyov
Tal Lavi
Ana Ostrovski
Michal Shteinberg
Michal Shteinberg
Galit Livnat
Galit Livnat
author_sort Karin Yaacoby-Bianu
collection DOAJ
description BackgroundCystic fibrosis (CF) is a chronic multi-systemic disease that requires a complex daily treatment regimen. Therefore, there is sub-optimal adherence to CF therapies, and it was shown to impact its clinical and economic burden. Cystic fibrosis transmembrane conductance regulator modulators (CFTRm) are high-cost medications that demonstrated significant benefit in clinical trials. The aim of this study was to evaluate the safety, usability, and efficacy of the ReX platform in medication management of CFTRm for the treatment of people with CF (pwCF).MethodsReX is a patient engagement platform consisting of a cloud-based management system and a cell-enabled handheld device intended to dispense oral medication into the patient's mouth, following a pre-programmed treatment protocol. It provides real-time adherence data to caregivers and timely, personalized reminders to patients. This is a prospective multi-center open study for pwCFs older than 12 years, who had been prescribed CFTRm [elexacaftor/tezacaftor/ivacaftor (ETI) or tezacaftor/ivacaftor (TI)], and provided consent to use ReX platform to receive CFTRm and record their health condition. Study duration was 12–24 months, with clinic visits where physical examination, body mass index (BMI), and pulmonary function tests were performed, and user experience questionnaires were filled in.ResultsTen pwCFs from two CF centers in Israel were included. The mean age was 31.5 years (range 15–74 years); eight were taking ETI and two TI. Median adherence to CFTRm was 97.5% (range 70%–100%) in the first year and 94% (range 84%–99%) in the second year, which is higher than the previously reported CFTRm adherence of ∼80%. No adverse events related to the use of the platform were reported. Patients reported ReX to be valuable to their treatment management and user friendly. Estimated mean forced expiratory volume in 1 s (FEV1%) increased from 74.4% to 80.8% (p = 0.004) over 2 years. Similarly, estimated BMI percentile increased from 53.5 to 59.0 (p < 0.001).ConclusionsUsing the ReX platform in medication management of pwCF treated by CFTRm is safe, easy to use, and effective in improving the adherence to treatment and the clinical outcomes. Consequently, this device may potentially reduce costs to healthcare providers. Further larger and long-term studies are required to examine the clinical benefits of the ReX platform.
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spelling doaj.art-61c75cf359804e9a891beb0489ed3b182023-12-19T08:54:51ZengFrontiers Media S.A.Frontiers in Pediatrics2296-23602023-12-011110.3389/fped.2023.13009681300968Optimizing CFTR modulator therapy management for cystic fibrosis through the ReX platformKarin Yaacoby-Bianu0Karin Yaacoby-Bianu1Malena Cohen-Cymberknoh2David Shoseyov3Tal Lavi4Ana Ostrovski5Michal Shteinberg6Michal Shteinberg7Galit Livnat8Galit Livnat9Pediatric Pulmonology Unit and CF Center, Carmel Medical Center, Haifa, IsraelB. Rappaport Faculty of Medicine, Technion—Israel Institute of Technology, Haifa, IsraelPediatric Pulmonology Unit and Cystic Fibrosis Center, Hadassah Medical Center and Faculty of Medicine, Hebrew University of Jerusalem, Jerusalem, IsraelPediatric Pulmonology Unit and Cystic Fibrosis Center, Hadassah Medical Center and Faculty of Medicine, Hebrew University of Jerusalem, Jerusalem, IsraelPharmacy Services, Carmel Medical Center, Haifa, IsraelPediatric Pulmonology Unit and CF Center, Carmel Medical Center, Haifa, IsraelPulmonology Institute and CF Center, Carmel Medical Center, Haifa, IsraelB. Rappaport Faculty of Medicine, Technion—Israel Institute of Technology, Haifa, IsraelPediatric Pulmonology Unit and CF Center, Carmel Medical Center, Haifa, IsraelB. Rappaport Faculty of Medicine, Technion—Israel Institute of Technology, Haifa, IsraelBackgroundCystic fibrosis (CF) is a chronic multi-systemic disease that requires a complex daily treatment regimen. Therefore, there is sub-optimal adherence to CF therapies, and it was shown to impact its clinical and economic burden. Cystic fibrosis transmembrane conductance regulator modulators (CFTRm) are high-cost medications that demonstrated significant benefit in clinical trials. The aim of this study was to evaluate the safety, usability, and efficacy of the ReX platform in medication management of CFTRm for the treatment of people with CF (pwCF).MethodsReX is a patient engagement platform consisting of a cloud-based management system and a cell-enabled handheld device intended to dispense oral medication into the patient's mouth, following a pre-programmed treatment protocol. It provides real-time adherence data to caregivers and timely, personalized reminders to patients. This is a prospective multi-center open study for pwCFs older than 12 years, who had been prescribed CFTRm [elexacaftor/tezacaftor/ivacaftor (ETI) or tezacaftor/ivacaftor (TI)], and provided consent to use ReX platform to receive CFTRm and record their health condition. Study duration was 12–24 months, with clinic visits where physical examination, body mass index (BMI), and pulmonary function tests were performed, and user experience questionnaires were filled in.ResultsTen pwCFs from two CF centers in Israel were included. The mean age was 31.5 years (range 15–74 years); eight were taking ETI and two TI. Median adherence to CFTRm was 97.5% (range 70%–100%) in the first year and 94% (range 84%–99%) in the second year, which is higher than the previously reported CFTRm adherence of ∼80%. No adverse events related to the use of the platform were reported. Patients reported ReX to be valuable to their treatment management and user friendly. Estimated mean forced expiratory volume in 1 s (FEV1%) increased from 74.4% to 80.8% (p = 0.004) over 2 years. Similarly, estimated BMI percentile increased from 53.5 to 59.0 (p < 0.001).ConclusionsUsing the ReX platform in medication management of pwCF treated by CFTRm is safe, easy to use, and effective in improving the adherence to treatment and the clinical outcomes. Consequently, this device may potentially reduce costs to healthcare providers. Further larger and long-term studies are required to examine the clinical benefits of the ReX platform.https://www.frontiersin.org/articles/10.3389/fped.2023.1300968/fullcystic fibrosisCFTR modulatorselexacaftor/tezacaftor/ivacaftormedication delivery platformsadherence to chronic treatmentremote patient monitoring
spellingShingle Karin Yaacoby-Bianu
Karin Yaacoby-Bianu
Malena Cohen-Cymberknoh
David Shoseyov
Tal Lavi
Ana Ostrovski
Michal Shteinberg
Michal Shteinberg
Galit Livnat
Galit Livnat
Optimizing CFTR modulator therapy management for cystic fibrosis through the ReX platform
Frontiers in Pediatrics
cystic fibrosis
CFTR modulators
elexacaftor/tezacaftor/ivacaftor
medication delivery platforms
adherence to chronic treatment
remote patient monitoring
title Optimizing CFTR modulator therapy management for cystic fibrosis through the ReX platform
title_full Optimizing CFTR modulator therapy management for cystic fibrosis through the ReX platform
title_fullStr Optimizing CFTR modulator therapy management for cystic fibrosis through the ReX platform
title_full_unstemmed Optimizing CFTR modulator therapy management for cystic fibrosis through the ReX platform
title_short Optimizing CFTR modulator therapy management for cystic fibrosis through the ReX platform
title_sort optimizing cftr modulator therapy management for cystic fibrosis through the rex platform
topic cystic fibrosis
CFTR modulators
elexacaftor/tezacaftor/ivacaftor
medication delivery platforms
adherence to chronic treatment
remote patient monitoring
url https://www.frontiersin.org/articles/10.3389/fped.2023.1300968/full
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