Psychometric Evaluation and Estimation of Meaningful Change Thresholds of Patient-Reported Outcome Measures in Chronic Inducible Cold Urticaria

Speaker(s)

Rhoten S1, Guillemin I2, Ivanescu C3, Oliván R4, Jingdong C5, Msihid J6, Martincova R7, Zikos E6, Krol M8, Chuang CC9, Brookes E10
1IQVIA, Patient Centered Solutions, San Francisco, CA, USA, 2IQVIA, Patient Centered Solutions, Lyon, France, 3IQVIA, Patient Centered Solutions, Amsterdam, Netherlands, 4IQVIA, Patient Centered Solutions, Barcelona, Spain, 5Regeneron Pharmaceuticals, Inc, Tarrytown, NY, USA, 6Sanofi, Gentilly, France, 7Sanofi, Prague, Czech Republic, 8IQVIA, Amsterdam, Netherlands, 9Sanofi, Cambridge , MA, USA, 10Sanofi, Reading, UK

Presentation Documents

OBJECTIVES: To assess the psychometric properties, including Meaningful Change Thresholds (MCTs) estimation of three patient-reported outcome measures (PROMs): Urticaria Control Test (UCT), Cold Urticaria Activity Score (ColdUAS), and Dermatology Life Quality Index (DLQI), in patients with chronic inducible cold urticaria (CICU).

METHODS: UCT comprised of 4-items yielding a total score (range 0–16, higher score: better disease control). ColdUAS is a 5-item PRO questionnaire yielding an average sum score (range 0–6, higher score: higher severity). DLQI comprised of 10-items yielding a total score (range 0–30, higher score: greater quality of life impairment). Data at baseline, Week-12, and Week-24 from a phase 3 dupilumab trial (NCT04681729) were analyzed to investigate reliability (internal consistency and test-retest), construct validity (convergent validity and known-groups validity), and sensitivity to change. Anchor-based approach was used to estimate within-patient and between-group MCTs using Patient Global Impression of Severity, Patient Global Impression of Change, and ColdUAS item-5 as anchors.

RESULTS: Baseline mean scores were 6.24 for UCT, 2.68 for ColdUAS, and 9.51 for DLQI. Adequate test-retest reliability for UCT (intraclass correlation coefficient range: 0.75–0.90) and ColdUAS (0.71–0.86), and low reliability for DLQI (0.04–0.51) were observed. Convergent validity was demonstrated by moderate-to-strong correlations for UCT and ColdUAS (absolute r range, 0.68–0.84 and 0.59–0.82, respectively), and low-to-strong correlations for DLQI (0.26–0.91). Adequate known-groups validity was demonstrated for all three PROMs, distinguishing between severity level groups. Significant differences in mean score changes over time were observed for groups defined using target anchors (all p <0.05). MCTs were estimated for within-patient meaningful change (UCT: 4, range 3–5; ColdUAS: 1.6, range 1.2–2.3; DLQI: 7, range 4–10) and between-group meaningful change (UCT: 3.3, range 2.5–3.9; ColdUAS: 1.1, range 0.9–1.5; DLQI: 4.7, range 3.5–6.8).

CONCLUSIONS: The study supported the use of UCT, ColdUAS, and DLQI in capturing patient experiences of CICU.

Code

PCR10

Topic

Patient-Centered Research

Topic Subcategory

Instrument Development, Validation, & Translation

Disease

Biologics & Biosimilars, Sensory System Disorders (Ear, Eye, Dental, Skin)