The Evaluation of Health-Related Quality of Life Issues Experienced by Patients with Desmoid-Type Fibromatosis (The QUALIFIED Study)-A Protocol for an International Cohort Study.
Sporadic desmoid-type fibromatosis (DTF) is a rare soft tissue tumour with an unpredictable clinical course. These tumours are incapable of metastasising, but their local aggressive tumour growth and tendency to recur locally can result in a substantial symptom burden. Measuring the impact of DTF on health-related quality of life (HRQoL) can be challenging due to the variable clinical presentation of the disease. Therefore, a HRQoL instrument assessing DTF-specific issues is needed. The QUALIFIED study aims to (1) pre-test a previously developed DTF-specific HRQoL tool (the DTF-QoL); (2) evaluate prevalence of HRQoL issues in adult DTF patients; and (3) identify subgroups at risk of impaired HRQoL. This study (NCT04289077) is an international, multicentre, cross-sectional, observational cohort study. Patients ≥ 18 years with sporadic DTF from the Netherlands and the United Kingdom will be invited to complete a set of questionnaires specifically composed for this patient group. Questionnaires will be completed using PROFILES (Patient Reported Outcomes Following Initial treatment and Long-term Evaluation of Survivorship). Analyses will include testing the psychometric properties of the DTF-QoL and evaluating the prevalence of HRQoL issues using the DTF-QoL, EORTC QOL-C30 and EQ-5D-5L, among other questionnaires. This study will provide insight into HRQoL issues experienced by patients with DTF. Awareness of these issues and the implementation of the DTF-QoL in research and clinical practice can help to improve overall HRQoL and to provide personalised care.
['Schut|Anne-Rose W|AW|', 'Timbergen|Milea J M|MJM|', 'Lidington|Emma|E|0000-0001-6176-8054', 'Grünhagen|Dirk J|DJ|0000-0001-8293-6002', 'van der Graaf|Winette T A|WTA|0000-0001-7549-3338', 'Sleijfer|Stefan|S|', 'van Houdt|Winan J|WJ|0000-0002-2303-3468', 'Bonenkamp|Johannes J|JJ|', 'Younger|Eugenie|E|0000-0002-2088-3328', 'Dunlop|Alison|A|', 'Jones|Robin L|RL|0000-0003-4173-3844', 'Verhoef|Cornelis|C|0000-0001-9980-8613', 'Gennatas|Spyridon|S|0000-0002-5635-7696', 'Husson|Olga|O|0000-0002-1387-8686']
health-related quality of life desmoid-type fibromatosis symptom burden symptoms disease-specific measures patient-reported outcomes rare diseases