Changes in symptom scores as a potential clinical endpoint for studies of cystic fibrosis pulmonary exacerbation treatment

J Cyst Fibros. 2021 Jan;20(1):36-38. doi: 10.1016/j.jcf.2020.08.006. Epub 2020 Aug 13.

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.

Publication types

  • Observational Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Anti-Bacterial Agents / therapeutic use*
  • Chronic Disease
  • Clinical Trials as Topic
  • Cystic Fibrosis / complications*
  • Disease Progression*
  • Humans
  • Respiratory Tract Infections / complications*
  • Respiratory Tract Infections / diagnosis
  • Respiratory Tract Infections / drug therapy*
  • Symptom Assessment / methods*
  • Young Adult

Substances

  • Anti-Bacterial Agents

Associated data

  • ClinicalTrials.gov/NCT02109822