Alexandra Gilbert

Use of patient-reported outcomes to measure symptoms and health related quality of life in the clinic

There is increasing interest in the use of patient-reported outcomes (PROs) in routine practice in cancer care to measure symptoms and health related quality of life (HRQOL). PROs are designed to capture the patients perspective of their care and treatment, and complement the traditional clinical outcomes of survival and toxicity assessment. Integrating routine collection and feedback of PROs has been found to improve care for patients on both an individual level, through improved communication and management of symptoms, and at an organizational level, by enabling aggregation of data to compare performance. This article reviews the benefits and challenges of introducing patient-reported assessments into routine clinical practice. Methods for choosing a questionnaire; collection and presentation of results; timing and frequency of administration as well as clinician training methods to aid the ability of clinicians to integrate the use of PROs into their own practice are described. Electronic PRO capture and integration with electronic health records seems to provide the most effective method for seamless integration into existing patient care pathways. Case studies from our own practice illustrate the issues raised. Electronic methods enabling immediate collection, scoring and interpretation of the data, as well as real-time data capture, email alert systems and individualized, online self-management advice may enable severe symptoms to be managed in a more timely manner. Evaluation methods are described to establish the effectiveness of the PRO intervention. Engaging stakeholders throughout the process of initial consultation and development, during delivery and evaluation is key to success. Future work needs to focus on the effectiveness of PROs in longer-term follow-up of patients in routine care and the relationship between the PRO severity grading and clinician severity grading using the Common Terminology Criteria of Adverse Events (CTCAE).

Patient-reported outcomes in randomised controlled trials of gynaecological cancers: investigating methodological quality and impact on clinical decision-making.

AIM The aim for this study is to investigate the methodological quality and potential impact on clinical decision making of patient reported outcome (PRO) assessment in randomised controlled trials (RCTs) in the gynaecological cancer sites. METHODS A systematic review identified RCTs published between January 2004 and June 2012. Relevant studies were evaluated using a pre-determined extraction form which included: (1) Trial demographics and clinical and PRO characteristics; (2) level of PRO reporting and (3) bias, assessed using the Cochrane Risk of Bias tool. All studies were additionally analysed in relation to their relevance in supporting clinical decision making. RESULTS Fifty RCTs enrolling 24,991 patients were identified. In eight RCTs (16%) a PRO was the primary end-point. Twenty-one studies (42%) were carried out in a multi-national context. Where statistically significant PRO differences between treatments were found, it related in most cases to both symptoms and domains other than symptoms (n=17, 57%). The majority of studies (n=42, 84%) did not mention the mode of administration nor the methods of collecting PRO data. Statistical approaches for dealing with missing data were only explicitly mentioned in nine RCTs (18%). Sixteen RCTs (32%) were considered to be of high-quality and thus able to inform clinical decision making. Higher-quality PRO studies were generally associated with RCTs that were at a low risk of bias. CONCLUSION This study showed that RCTs with PROs were generally well designed and conducted. In a third the information was very informative to fully understand the pros and cons of PROs treatment decision-making.

Choice of a patient-reported outcome measure for patients with anal cancer for use in cancer clinical trials and routine clinical practice: a mixed methods approach

BACKGROUND In the USA, more than 7200 new cases of anal cancer were diagnosed in 2014 with incidence rising. Concurrent chemoradiotherapy improves cancer-related outcomes but has led to an increase in acute and late adverse events. Patient-reported outcomes (PRO) are increasingly included in trials as a surrogate measure for reporting of late adverse events. This study aimed to select the most effective PRO to use in clinical research and practice for anal cancer since no questionnaire specific for anal cancer has been developed. METHODS A mixed methods approached was used. A systematic review estimated the frequency of use of PROs and clinician reporting instruments used in anal cancer trials including radiation treatment. Health professional semi-structured interviews explored preferred questionnaires and revealed missing items; analysis was done using the framework approach. Two symptom-based, validated questionnaires were selected and assessed by means of inductive content analysis to highlight discrepancies relevant to anal cancer. FINDINGS 34 relevant studies reported on radiotherapy adverse events. For PROs, EORTC QLQ-C30 (n=5) and EORTC QLQ-CR38 (n=3) were used most frequently. RTOG/EORTC (n=17) and CTCAE (n=15) criteria were most commonly used for clinician reporting. EORTC QLQ-C30 and EORTC QLQ-CR38, and National Cancer Institutes (NCI) PRO-CTCAE were selected for further analysis. No consensus for questionnaire content or design was found through health professional interviews (n=8). Domains and codes relevant to anal cancer treatment were selected from interviews to inform questionnaire analysis. 27 domains and 60 codes were found in EORTC questionnaires, and 21 domains and 44 codes in NCI PRO-CTCAE. Four domains and 16 codes were not covered by EORTC systems including radiation skin reaction. Six domains and 45 codes were not covered by NCI PRO-CTCAE including vaginal stenosis and bowel urgency. INTERPRETATION This study shows that there are gaps in the questionnaires commonly used to record adverse events using PROs for anal cancer. Expert opinion is valuable in highlighting relevant missing items but provides no consensus on design and wording preferences. For use in longitudinal follow-up of patients with anal cancer treated with (chemo)radiation, the EORTC-QLQ system has the fewest missing symptom items for use as a validated PRO in clinical research and practice. FUNDING This abstract presents independent research funded by the National Institute for Health Research (NIHR).