Assessment of health-related quality of life and patient benefit as outcome measures for clinical trials in hepatocellular carcinoma

Aim: The purpose of the study was to determine the feasibility and validity of using health-related quality of life (HRQL) and patient benefit as outcome measures in a phase II trial of octreotide long acting release (LAR) for hepatocellular carcinoma (HCC); and, to determine aspects of HRQL that were most impaired and aspects that changed. Methods: Sixty-three patients with advanced HCC were treated with octreotide LAR. HRQL was assessed monthly with the Functional Assessment of Cancer Therapy - Hepatobiliary Questionnaire (FACT-Hep) and the Patient Disease and Treatment Assessment Form (Patient DATA Form). Benefit was assessed directly by asking subjects at 1 month about change from baseline using the Patient Benefit Form. Convergent, discriminative and predictive validity were assessed by testing a priori hypotheses. Results: Seventy-three percent completed a baseline assessment of HRQL. Compliance decreased over time. Fatigue, energy, anxiety, pain, insomnia, and emotional well-being were the most impaired aspects of HRQL on the Patient DATA Form. Convergent validity was supported by 52 of 57 hypothesized correlations. Predictive validity was supported by associations between survival and four aspects of HRQL. Patients' direct assessments of benefit for common symptoms were moderately associated with changes in their scores on the Patient DATA Form. Conclusion: Use of the FACT Hep and Patient DATA Form is feasible in trials of advanced HCC. The validity of the direct measure of patient benefit was questionable, despite its intuitive attraction. Fatigue, anxiety, insomnia, and impaired emotional well-being were prominent, are probably under-recognized in practice, and are worthy of future research.