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.

Leopold Hentschel,Ioanna Nixon,Ingrid M.e Desar ,Dide Den Hollander,Martin Eichler,Aisha Miah,Javier Martín-Broto ,Bernd Kasper,Samantha Sodergren ,Susanne Schnittger ,Olga Husson,Antonio Casado Herráez ,Winette T.a Van Der Graaf ,Hanna Koseła-Paterczyk ,Winan J Van Houdt,Claire Piccinin,Isabelle Ray‐Coquard ,Roger Wilson,Marco Fiore,Danijela Kuliš

doi:10.3390/cancers13010001

Abstract

Simple SummarySarcomas 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.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.

Highlights

Sarcomas are a group of rare malignant solid neoplasms of mesenchymal origin, comprising more than 100 histological and more than 250 molecular subtypes [1]
This study aims to develop an exhaustive list of all health-related quality of life (HRQoL) issues relevant to sarcoma patients and to determine content validity
In order to facilitate transparent decision-making concerning the best approach to take in developing a sarcoma measurement strategy, the existing decision criteria will be followed: (1) an inclusive approach will be taken with regard to relevant items; (2) inclusion of an issue will be considered when more than 30% of patients mention the issue as missing; (3) issues that have a low mean score for relevance or importance will be considered for exclusion

Summary

Introduction

Sarcomas are a group of rare malignant solid neoplasms of mesenchymal origin, comprising more than 100 histological and more than 250 molecular subtypes [1]. Significant heterogeneity exists among cases with widely different patterns of the stage at diagnosis, tumor location at almost any anatomical site, prognosis, treatments, and age at diagnosis [2]. The overall incidence of sarcoma is about 7 in 100,000 persons, with 30,000 new cases a year in Europe [3]. 10% of patients present with de novo metastatic disease. Many sarcomas have aggressive biological behavior, and around half of patients with high-grade tumors will eventually develop incurable metastatic disease [4,5]. The five-year relative survival in Europe (period 2000–2007) is 60% for STS and 50% for BS [6]; survival in the metastatic setting is poor; for example, in STS, median overall survival is

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