Psychometric Analysis of the EORTC QLQ-NMIBC24 Using Data from a Global Phase 2 Randomized Trial of Erdafitinib[1] in Participants with High-Risk Non-Muscle-Invasive Bladder Cancer (NMIBC)
Author(s)
Kristi Bertzos, PhD1, Carrie Houts, PhD2, André De Champlain, PhD1, Anthony Raborn, PhD2;
1Janssen Global Services, LLC, Horsham, PA, USA, 2Vector Psychometric Group, LLC, Chapel Hill, NC, USA
1Janssen Global Services, LLC, Horsham, PA, USA, 2Vector Psychometric Group, LLC, Chapel Hill, NC, USA
Presentation Documents
OBJECTIVES: The European Organization for Research and Treatment of Cancer (EORTC) QLQ-NMIBC24 is a disease-specific, PROM designed to supplement the EORTC QLQ-C30 when evaluating health-related quality of life in adults with non-muscle-invasive bladder cancer (NMIBC). Although evidence supports the measurement properties of the QLQ-NMIBC24 (e.g., Blazeby et al., 2014), it has not been field-tested in a global population. The objective of this research was to evaluate the psychometric properties of the QLQ-NMIBC24 in a global sample of NMIBC patients.
METHODS: A randomized, Phase 2 trial enrolled 107 adults with intermediate- or high-risk NMIBC in 15 countries. Analyses of the QLQ-NMIBC24 focused on data from screening and the first three treatment cycles. Dimensionality of the QLQ-NMIBC24 items was assessed using factor analysis. Classical test theory (CTT) methods assessed internal consistency and test-retest reliability. Validity was assessed by convergent and discriminant correlational, known-groups, and sensitivity to change analyses. Meaningful score difference (MSD) thresholds were also investigated.
RESULTS: A unidimensional factor model applied to the Urinary Symptoms domain showed adequate fit (TLI = 1.00, RMSEA = 0.04) after accounting for residual correlations between two items. CTT analyses showed high internal consistency for both the Urinary Symptoms (alpha = 0.83) and Future Worries (alpha = 0.86) domains. All domains displayed acceptable test-retest reliability in a stable sample of participants, except for Malaise (ICC = .19). Different methods of validation analyses showed that the QLQ-NMBIC24 scores generally behaved as expected. Distribution-based MSD thresholds were around 10 points for all but the Male Sexual Problems domain.
CONCLUSIONS: This is the first investigation of the psychometric properties and MSD thresholds for the QLQ-NMIBC24 in a global sample. Results provide additional support for the reliability and validity of inferences that can be made using QLQ-NMIBC24 domain scores in adults living with NMIBC.
[1] Erdafitinib (JNJ-42756493) was discovered in collaboration with Astex Pharmaceuticals
METHODS: A randomized, Phase 2 trial enrolled 107 adults with intermediate- or high-risk NMIBC in 15 countries. Analyses of the QLQ-NMIBC24 focused on data from screening and the first three treatment cycles. Dimensionality of the QLQ-NMIBC24 items was assessed using factor analysis. Classical test theory (CTT) methods assessed internal consistency and test-retest reliability. Validity was assessed by convergent and discriminant correlational, known-groups, and sensitivity to change analyses. Meaningful score difference (MSD) thresholds were also investigated.
RESULTS: A unidimensional factor model applied to the Urinary Symptoms domain showed adequate fit (TLI = 1.00, RMSEA = 0.04) after accounting for residual correlations between two items. CTT analyses showed high internal consistency for both the Urinary Symptoms (alpha = 0.83) and Future Worries (alpha = 0.86) domains. All domains displayed acceptable test-retest reliability in a stable sample of participants, except for Malaise (ICC = .19). Different methods of validation analyses showed that the QLQ-NMBIC24 scores generally behaved as expected. Distribution-based MSD thresholds were around 10 points for all but the Male Sexual Problems domain.
CONCLUSIONS: This is the first investigation of the psychometric properties and MSD thresholds for the QLQ-NMIBC24 in a global sample. Results provide additional support for the reliability and validity of inferences that can be made using QLQ-NMIBC24 domain scores in adults living with NMIBC.
[1] Erdafitinib (JNJ-42756493) was discovered in collaboration with Astex Pharmaceuticals
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
CO147
Topic
Clinical Outcomes
Topic Subcategory
Clinical Outcomes Assessment
Disease
SDC: Oncology