Background: CFTR modulator therapy initially with Ivacaftor and now with Elexacaftor-Tezacaftor-Ivacaftor triple therapy has revolutionised therapy for Cystic Fibrosis. We wanted to look at the real-world outcomes of our patients transitioning to triple therapy over a 12-month period. We followed them in the clinic and virtually using a smartphone app that allowed integration of patient recorded outcomes and objective data like spirometry and weight uploaded to a web-based portal.
Methods: The study recruited 18 patients about to start on triple therapy. Initially patients were brought into the clinic and baseline data including height, weight, lung function, sputum volume, sputum culture and routine bloods were collected. Initial data was recorded on an app (PatientMpower) on the patient’s smartphone including sputum volume and quality of life questionnaire’s. A weighing scales and Spirometer were connected to the smartphone app via Bluetooth. At 3 month intervals a virtual consultation took place with our study co-ordinator. Weight and lung function were performed and downloaded to the app. Lung function was assessed using the Mir Spirobank Smart Handheld spirometer. Lung function was also formally measured in the lab at baseline and between 6 and 9 months. Weight was recorded at the patient’s home using a bodytrace weighing scales, and the results were transmitted to the patientmpower app. Sputum volume was recorded on the app by the patient at baseline and at 3 monthly intervals. The Cystic Fibrosis Respiratory Symptom Diary (CFRSD) was recorded on the app and the CFQ Resp was also completed.
Results: The number of patients that completed the study was 18, 4 female and 14 male patients were included in the analysis. 15 patients were homozygous for F508del and 3 patients were heterozygous F508del and therefore naive to CFTR modulators. The mean (SD) absolute increase in the ppFEV1 was +15.05 (P < 0.00001). Weight also increased significantly by up to 4.22kg (p < 0.0001). The mean reduction in sputum volume per day was 27.5mls (17.38). At baseline more than 50% of patients had sputum production in excess of 30mls/day, but after initiation of CFTR triple therapy sputum production fell to much lower volumes for at least 50% of patients in the study and maintained at negligible levels through 12 months. The mean improvement in CFQ-R was 17.6 (12.8) points. The CFRSD questionnaire also showed significant improvement falling by 20.3 (11.9) points where a change of 4 points is considered to be clinically significant.
Conclusions: The excellent results seen in our study mirror those of the phase III clinical trials. The reduction in sputum volume while subjective was impressive and hasn't been reported quantitatively before to our knowledge. The smartphone app was particularly useful in providing accurate data remotely and would allow for more frequent and convenient opportunities to capture clinical data from patients without having to come to the study centre.
Acknowledgements: This work was supported by funding from Sláintecare. .
