Abstract
To demonstrate the measurement properties of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Hepatocellular Carcinoma 18-question module (EORTC QLQ-HCC18) within a previously treated, unresectable hepatocellular carcinoma (HCC) clinical trial population that was distinct from the published QLQ-HCC18 validation population. Analyses were conducted using data from BGB-A317-208, an open label, international, clinical trial assessing efficacy and safety of the monoclonal antibody tislelizumab in adult HCC patients. The EORTC Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) and QLQ-HCC18 instruments were assessed at baseline and weeks 3 and 9 follow-up visits. Per US Food and Drug Administration guidance, psychometric validation of the QLQ-HCC18 included reliability (internal consistency and test–retest), construct validity (convergent and discriminant validity and known-groups validity), ability to detect change, and meaningful within-patient change (MWPC). Known-groups validity and MWPC analyses were also stratified on several pre-defined subgroups. A total of 248 patients were included. Only the QLQ-HCC18 fatigue, nutrition, and index domains demonstrated acceptable internal consistency; acceptable test–retest reliability was found for fatigue, body image, nutrition, pain, sexual interest, and index domains. The QLQ-HCC18 fatigue domain achieved the pre-specified criterion defining acceptable convergent and discriminant validity for 13 of 16 correlations, whereas the index domain achieved the pre-specified criterion for 14 of 16 correlations. Clear differentiation of the QLQ-HCC18 change scores between improvement and maintenance anchor groups were observed for body image, fatigue, pain, and index domains, whereas differentiation between deterioration and maintenance anchor groups were observed for fever and fatigue domains. MWPC point estimates defining improvement for the QLQ-HCC18 fatigue and index domains were −7.18 and −4.07, respectively; MWPC point estimates defining deterioration were 5.34 and 3.16, respectively. The EORTC QLQ-HCC18 fatigue and index domains consistently demonstrated robust psychometric properties, supporting the use of these domains as suitable patient-reported endpoints within a previously treated, unresectable HCC patient population.
Highlights
Hepatocellular carcinoma (HCC) is a substantial global health challenge that accounts for 85% to 90% of all reported cases of liver cancer and is the fourth most common cause of cancer-related death [1]
This study was conducted in accordance with sponsor procedures, which comply with the principles of Good Clinical Practice, International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use Guidelines, the Declaration of Helsinki, and local regulatory requirements
A sample size of 228 was calculated to provide a power of 0.97 to demonstrate that the objective response rate in patients with previously treated unresectable HCC is statistically higher than the historical rate of 7% in a binomial exact test at a one-sided alpha level of 0.025
Summary
Hepatocellular carcinoma (HCC) is a substantial global health challenge that accounts for 85% to 90% of all reported cases of liver cancer and is the fourth most common cause of cancer-related death [1]. The majority of HCC cases (> 80%) occur in Eastern Asia and sub-Saharan Africa, with typical incidence rates of > 20 per 100,000 individuals: China alone accounts for approximately 50% of both new HCC cases and HCC-related deaths worldwide [4, 5]. Patients with unresectable HCC represent a population with great unmet medical need, having a 5-year overall survival (OS) rate of 18% [6].
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