Patient-reported quality of life and symptom burden measures in human papillomavirus associated oropharyngeal cancer – A review of the literature and PRO methodology. (July 2021)

• Record Type: Journal Article
• Authors: McDowell, Lachlan; Gough, Karla; King, Madeleine; Corry, June; Rischin, Danny
• Is Part Of: Oral oncology. Volume 118(2021)
• Publication Date: 2021-07
• Subjects: Oropharyngeal neoplasms -- Quality of life -- Survivorship -- Robotic surgical procedures -- Minimal clinically important difference -- Radiotherapy -- Proton therapy -- Chemotherapy -- Psychometrics -- Human Papillomavirus Infection; Mouth -- Cancer -- Periodicals; Mouth -- Tumors -- Periodicals; Mouth Diseases -- Periodicals; Mouth Neoplasms -- Periodicals; Bouche -- Cancer -- Périodiques; Bouche -- Tumeurs -- Périodiques; Tumeurs -- Périodiques; Electronic journals; 616.9943105
• DOI: 10.1016/j.oraloncology.2021.105309
• Languages: English
• ISSNs: 1368-8375

Abstract

Highlights: PROs are the core of determining the optimal treatment approach for patients with HPV OPC. PROs must be applied with scientific rigor in order to answer current research questions. PROs should be interpreted in the context of clinical significance. Improving our understanding of the psychometric properties of PROs is needed. Our specialty needs to increase the sophistication with which PROs are analysed and interpreted. Abstract: The emergence of human papillomavirus-associated oropharyngeal cancer (HPVOPC) has resulted in an explosion of clinical research offering reduced toxicity and improved health-related quality of life (HRQL) through treatment de-escalation. At the heart of this objective are patient-reported outcomes (PROs) which aim to quantify the patient experience, usually through the measurement of HRQL or symptom burden. A number of PRO measures (PROMs) are available to HNC researchers and selection of the optimal instrument relies on a detailed understanding of their content and psychometric properties matched to the clinical endpoint of interest. As PROMs become increasingly favoured as the primary or co-primary endpoints of interest in HNC clinical trials, particularly those focussed on HPVOPC, future treatment paradigms will be determined by these measures and it is imperative that they are applied with sophistication and rigor. This review draws attention to the limitations and challenges our specialty faces in PRO application, analysis and reporting. These shortfalls typically include a reliance on statistical rather than clinically relevant differences, multiple hypothesis testing, a lack of evidence-based minimal clinically important differences for the commonly used tools, as well as variations in PROM selection. The aim of this review is to provide: (1) an overview of PRO/PROM terminology and methodology in the HNC setting; (2) to provide a summary of HRQL and symptom burden reports in the HPVOPC literature; and (3) to draw attention to the unmet research need of refining PROM development, application and interpretation to guide our treatment decisions based on what matters to patients.