Measuring and Reporting Health-Related Quality of Life (HRQoL) in Locally Advanced/Metastatic Urothelial Cancer (La/mUC) Research: Capturing Outcomes That Really Matter to Patients
Author(s)
Mairead Kearney, MB BCh, MPH, MBA, MSc. Econ1, Thomas Macmillan, BA2, Julia Poritz, PhD2, Sherrie Schreiber-Gosche, MSc2, Mihaela G. Musat, PhD2;
1the healthcare business of Merck KGaA, Darmstadt, Germany, 2Cytel, Cambridge, MA, USA
1the healthcare business of Merck KGaA, Darmstadt, Germany, 2Cytel, Cambridge, MA, USA
Presentation Documents
OBJECTIVES: Despite growing emphasis on maintaining HRQOL in la/mUC, it remains unclear if HRQOL instruments address the specific dimensions of health most important to patients or if HRQOL data are adequately reported. We evaluated how thoroughly HRQOL instruments measure symptoms reported by patients with la/mUC and the quality with which HRQOL results are reported.
METHODS: A systematic literature review (SLR) was conducted to identify clinical trials and real-world evidence (RWE) reporting HRQOL outcomes in la/mUC published before May 29, 2024. Qualitative research was identified via a targeted literature review (TLR; August 2024). The CONSORT-PRO checklist was used to evaluate HRQOL reporting.
RESULTS: The SLR identified 49 studies (37 clinical trials, 12 RWE studies); the TLR identified 5 qualitative studies. Since 2000, EORTC QLQ-C30 was the most frequently used instrument, while newer disease-specific instruments were used in more recent years. Based on qualitative evidence, patients’ concerns included pain, fatigue, hematuria, other urinary symptoms, sleep disturbance, sexual dysfunction, depression/anxiety/mental well-being, nausea/vomiting, hair loss, weight loss, and appetite loss. Coverage of these items is better in the newer disease-specific instruments (FACT-Bl, 82%; NFBlSI-18, 77%) than in generic instruments like the SF-36 (27%). Coverage is moderate for oncology-specific measures (EORTC QLQ-C30, 55%; FACT-G, 55%). Hematuria and hair loss were not addressed by any instrument, and the NFBlSI-18 item assessing sexual dysfunction applies to men only. Per the CONSORT-PRO checklist, HRQOL outcomes reporting is generally poor. Not all studies reported baseline and follow-up data, and <50% provided background, rationale, or hypotheses for analyses.
CONCLUSIONS: HRQOL instruments currently used in la/mUC research do not adequately capture patient concerns or symptoms, and findings are not consistently reported in a transparent and comprehensive manner. Future recommendations include using PRO-CTCAE items to supplement existing HRQOL instruments and better adherence to reporting guidelines.
METHODS: A systematic literature review (SLR) was conducted to identify clinical trials and real-world evidence (RWE) reporting HRQOL outcomes in la/mUC published before May 29, 2024. Qualitative research was identified via a targeted literature review (TLR; August 2024). The CONSORT-PRO checklist was used to evaluate HRQOL reporting.
RESULTS: The SLR identified 49 studies (37 clinical trials, 12 RWE studies); the TLR identified 5 qualitative studies. Since 2000, EORTC QLQ-C30 was the most frequently used instrument, while newer disease-specific instruments were used in more recent years. Based on qualitative evidence, patients’ concerns included pain, fatigue, hematuria, other urinary symptoms, sleep disturbance, sexual dysfunction, depression/anxiety/mental well-being, nausea/vomiting, hair loss, weight loss, and appetite loss. Coverage of these items is better in the newer disease-specific instruments (FACT-Bl, 82%; NFBlSI-18, 77%) than in generic instruments like the SF-36 (27%). Coverage is moderate for oncology-specific measures (EORTC QLQ-C30, 55%; FACT-G, 55%). Hematuria and hair loss were not addressed by any instrument, and the NFBlSI-18 item assessing sexual dysfunction applies to men only. Per the CONSORT-PRO checklist, HRQOL outcomes reporting is generally poor. Not all studies reported baseline and follow-up data, and <50% provided background, rationale, or hypotheses for analyses.
CONCLUSIONS: HRQOL instruments currently used in la/mUC research do not adequately capture patient concerns or symptoms, and findings are not consistently reported in a transparent and comprehensive manner. Future recommendations include using PRO-CTCAE items to supplement existing HRQOL instruments and better adherence to reporting guidelines.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
PCR184
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Oncology