Changes in symptom scores as a potential clinical endpoint for studies of cystic fibrosis pulmonary exacerbation treatment
D R VanDevanter, S L Heltshe, D B Sanders, N E West, M Skalland, P A Flume, C H Goss, STOP-OB Study, D R VanDevanter, S L Heltshe, D B Sanders, N E West, M Skalland, P A Flume, C H Goss, STOP-OB Study
Abstract
Introduction: Symptom improvement was assessed as changes in the Chronic Respiratory Infection Symptom Score (CRISS) during intravenous antimicrobial exacerbation treatments among subjects from study NCT02109822.
Methods: Median daily CRISS reduction (i.e., improvement) and covariates associated with CRISS reduction by Day 14 were assessed by logistic regression.
Results: Among 173 subjects, median baseline CRISS was 49 [IQR 41, 56]; 93.6% had a CRISS reduction of ≥11 (minimal clinically important difference); median time to -11 reduction was 2 days [95% CI 2, 3]. The greatest median CRISS difference from baseline, on Day 17, was -26 [-29, -23]. Odds of -26 CRISS change by Day 14 were greater in subjects with higher baseline CRISS (P=.006) and younger ages (P=.041).
Conclusions: CRISS response has good dynamic range and may be a useful efficacy endpoint for PEx interventional trials. The optimal use of CRISS change as an endpoint remains uncharacterized.
Keywords: Clinical trials; Cystic fibrosis; Exacerbations; Symptoms.
Conflict of interest statement
Declaration of Competing Interest None
Copyright © 2020 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.
Figures
![Figure 1.. Notch-and-whisker plot of CRISS measure…](https://www.ncbi.nlm.nih.gov/pmc/articles/instance/8298137/bin/nihms-1718146-f0001.jpg)
![Figure 2.. Kaplan Meier curves of time…](https://www.ncbi.nlm.nih.gov/pmc/articles/instance/8298137/bin/nihms-1718146-f0002.jpg)
![Figure 3.. CRISS values at baseline, Day…](https://www.ncbi.nlm.nih.gov/pmc/articles/instance/8298137/bin/nihms-1718146-f0003.jpg)
Source: PubMed