Physical symptom burden in patients with desmoid‐type fibromatosis and its impact on health‐related quality of life and healthcare use

• Published in: Cancer medicine (Malden, MA) Vol. 12; no. 12; pp. 13661 - 13674
• Main Authors: Schut, Anne‐Rose W., Bruin, Leanne E., Rooij, Belle H., Lidington, Emma, Timbergen, Milea J. M., Graaf, Winette T. A., Houdt, Winan J., Bonenkamp, Johannes J., Jones, Robin L., Grünhagen, Dirk. J., Sleijfer, Stefan, Gennatas, Spyridon, Verhoef, Cornelis, Husson, Olga
• Format: Journal Article
• Language: English
• Published: United States John Wiley & Sons, Inc 01.06.2023; John Wiley and Sons Inc; Wiley
• Subjects: Cancer therapies; Cluster analysis; Connective tissue diseases; Cross-Sectional Studies; Data collection; Delivery of Health Care; desmoid tumour; Fibromatosis, Aggressive - therapy; Health care; Health services utilization; healthcare utilisation; health‐related quality of life; Humans; Likert scale; Pain; Pain - etiology; Patients; patient‐reported outcomes; Quality of Life; Questionnaires; rare diseases; Regression analysis; Sociodemographics; Surveys and Questionnaires; Therapists; Therapy; Variables; Variance analysis; United Kingdom > UK; Netherlands; rare diseases; patient-reported outcomes; health-related quality of life; desmoid tumour; healthcare utilisation
• DOI: 10.1002/cam4.5985
• ISSN: 2045-7634

Background Desmoid‐type fibromatosis (DTF) has a highly variable clinical course with varying intensity of symptoms. The objectives of this study were to identify subgroups of DTF patients based on physical symptom burden and to compare symptom burden subgroups on health‐related quality of life (HRQoL) and healthcare use (univariate and multivariate). Methods Desmoid‐type fibromatosis patients from the United Kingdom and the Netherlands received cross‐sectional questionnaires on HRQoL (EORTC QLQ‐C30), DTF‐specific HRQoL (DTF‐QoL) and healthcare utilisation. Latent class cluster analysis was performed to identify subgroups based on patients' symptom burden using EORTC QLQ‐C30 and DTF‐QoL physical symptom items. Multivariate linear and logistic regression analyses were conducted to examine associations of symptom burden with HRQoL and healthcare utilisation, respectively. Results Among 235 DTF patients, four symptom burden clusters were identified, with low symptom burden (24%), intermediate symptom burden‐low pain (20%), intermediate symptom burden‐high pain (25%) and high symptom burden (31%). DTF patients with high symptom burden had clinically relevant lower HRQoL scores compared to patients with low and intermediate symptom burden (p < 0.001) and reported more general and DTF‐related visits to their general practitioner compared to the low symptom burden cluster (p < 0.01). In the multivariate analyses, symptom burden was independently associated with both HRQoL and healthcare utilisation. Conclusions This study identified four distinct subgroups of DTF patients based on their level of symptom burden, with a considerable number of patients being highly symptomatic. Knowledge of the level of symptom burden DTF patients experience can help to identify patients at risk of poorer outcomes and tailor supportive care to the individual needs of DTF patients.