This is a repository copy of Prognostic value of patient-reported outcomes from international randomised clinical trials on cancer: a systematic review.

Background: Considering the worldwide incidence of breast cancer (BC) and the importance of health-related quality of life (HRQoL) assessment, there is a growing need to have accurate and up-to-date reference values (RVs). RVs are useful for the design of randomised controlled trials (RCTs) and as benchmarks for comparison of cancer RCTs and health care interventions. This study aimed to provide RVs for the QLQ-C30 in early BC (EBC) and metastatic BC (MBC). General patterns of main results from the EORTC dataset (main dataset) were compared with the PDS dataset (comparison dataset) to see whether they would be consistent across pre-defined covariates.RCTs where baseline HRQoL (before treatment) was assessed with the QLQ-C30. RVs were calculated and stratified by disease stage, age, and when available, performance status (PS), comorbidity and region. RVs were reported using descriptive statistics. Results: Data from three EORTC (n Z 4115) and three PDS RCTs (n Z 1406) were included in the analysis. While EBC patients presented better HRQoL with high baseline functioning scores and low prevalence of symptoms, MBC patients reported worse HRQoL with lower functioning scores and more prevalence of symptoms. In MBC, poor PS and presence of comorbidities reflected worse baseline HRQoL. No consistent differences were found for age and countries. Conclusion: These up-to-date RVs for the EORTC QLQ-C30 in BC show differences in HRQoL scores between stages, PS, and comorbidities. These findings, supported by an independent dataset, will help the clinical interpretation of scores for BCpatients. ª

No abstract

Objectives To provide reference values for the European Organisation for Treatment and Research of Cancer (EORTC) Quality of Life Questionnaire (QLQ‐C30) in advanced‐stage Hodgkin lymphoma (HL) patients and 5‐year HL survivors. The QLQ‐C30 is the most widely used cancer‐specific questionnaire to assess Health‐Related Quality of Life (HRQoL). Methods The EORTC database was searched to identify HL RCTs in which patients’ and survivors’ HRQoL was assessed by the QLQ‐C30. HRQoL mean scores were calculated and stratified by age and gender. Minimal important differences were used to assess the clinical relevance of the findings. Data from one RCT with HRQoL scores available at baseline (n = 343) and four RCTs with HRQoL scores available at follow‐up (n = 1665) were analyzed. Results Patients reported worse HRQoL scores than survivors across most functioning scales and symptoms’ scales. These scores varied as a function of gender but not age. Survivors’ HRQoL reports were comparable to the ones of the general population. Conclusions These values provide an assessment framework for the comparison and interpretation of QLQ‐C30 scores in advanced‐stage HL. Our findings suggest that although HL patients’ HRQoL scores are worse than the general population, HRQoL scores may normalize over long‐term survival.

OBJECTIVES:PROs from open label trials are traditionally regarded as being more biased than PROs from double blinded studies, as the patient's knowledge of their treatment might influence their perception of their symptoms, functioning and HRQoL. We explored this hypothesis by comparing PROs from the QLQ-C30 which was used in patients treated with dabrafenib plus trametinib (D+T) for BRAF+ metastatic melanoma with the same data collection schedule in two separate studies e COMBI-D (double blinded vs dabrafenib, n¼211 in D+T arm) and COMBI-V (open label vs vemurafenib, n¼352 in D+T arm). METHODS: We qualitatively and quantitatively compared responses to the QLQ-C30 in the active dabrafenib+trametinib arms. Mean values and standard deviations for each QLQ-C30 domain were compared between trials at each time point until <20% of patients remained on trial. RESULTS: Qualitatively, there were no differences with graphs visually overlaying. This was consistent across all domains, including those showing clinically relevant improvements (HRQoL, pain) and those with no change. A small visual trend was observed in the COMBI-V open label study for constipation (worse in COMBI-V by mean of 2.0 points) and emotional functioning (better in COMBI-V by mean of 3.4 points) but not appetite loss, pain, dyspnea, fatigue, financial difficulties, insomnia, nausea and vomiting, physical functioning, role functioning, cognitive functioning or global health status/HRQoL. Quantitatively, in only 1 out of 98 timepoints, a difference larger than the MID of 5 was seen (emotional functioning, week 32, D5.4pts). CONCLUSIONS: This analysis demonstrates that there was little influence on the PRO results for the open label study COMBI-V versus COMBI-D a blinded study. The results were remarkably consistent qualitatively and numerically across these two independent studies. Future research is needed to validate this result across therapies and diseases to give decision makers confidence in interpreting open label PRO data.