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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Frontiers Media S.A.
2023-12-01
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Series: | Frontiers in Pediatrics |
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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. |
first_indexed | 2024-03-08T22:06:12Z |
format | Article |
id | doaj.art-61c75cf359804e9a891beb0489ed3b18 |
institution | Directory Open Access Journal |
issn | 2296-2360 |
language | English |
last_indexed | 2024-03-08T22:06:12Z |
publishDate | 2023-12-01 |
publisher | Frontiers Media S.A. |
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series | Frontiers in Pediatrics |
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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