Incorporating the Patient Voice in Sarcoma Research: How Can We Assess Health-Related Quality of Life in This Heterogeneous Group of Patients? A Study Protocol

SIMPLE SUMMARY: Sarcomas are a rare group of heterogeneous tumors. Because treatment side-effects detract from the survival benefit of treatment, it is important to assess treatment effectiveness in terms of patient-reported health-related quality of life (HRQoL). However, a sarcoma specific measurement instrument or strategy does currently not exist. This study aims to (1) develop a list of all HRQoL issues relevant to sarcoma patients; (2) determine a strategy for sarcoma-specific HRQoL assessment. An international, multicenter study will be conducted, searching existing literature and conducting interviews with adult sarcoma patients and healthcare professionals (HCPs) to create a list of HRQoL issues. Subsequently, another group of sarcoma patients and HCPs will be asked to rate and prioritize the issues. This information will help create a strategy to measure HRQoL in sarcoma patients, taking into account the heterogeneity of sarcomas, which will improve the collection of personalized patient-reported outcome data in future research and clinical care. ABSTRACT: Sarcomas comprise 1% of adult tumors and are very heterogeneous. Long-lasting and cumulative treatment side-effects detract from the (progression-free) survival benefit of treatment. Therefore, it is important to assess treatment effectiveness in terms of patient-reported outcomes (PROs), including health-related quality of life (HRQoL) as well. However, questionnaires capturing the unique issues of sarcoma patients are currently lacking. Given the heterogeneity of the disease, the development of such an instrument may be challenging. The study aims to (1) develop an exhaustive list of all HRQoL issues relevant to sarcoma patients and determine content validity; (2) determine a strategy for HRQoL measurement in sarcoma patients. We will conduct an international, multicenter, mixed-methods study (registered at clinicaltrials.gov: NCT04071704) among bone or soft tissue sarcoma patients ≥18 years, using EORTC Quality of Life Group questionnaire development guidelines. First, an exhaustive list of HRQoL issues will be generated, derived from literature and patient (n = 154) and healthcare professional (HCP) interviews (n = 30). Subsequently, another group of sarcoma patients (n = 475) and HCPs (n = 30) will be asked to rate and prioritize the issues. Responses will be analyzed by priority, prevalence and range of responses for each item. The outcome will be a framework for tailored HRQoL measurement in sarcoma patients, taking into account sociodemographic and clinical variables.