Patient reported outcomes in patients with desmoid type fibromatosis.

Abstract

11574 Background: Desmoid type fibromatosis (DTF) is a rare benign neoplasm with infiltrative growth and high local recurrences. Due to long disease course, unpredictable growth pattern, and low mortality, using only survival outcomes may be inappropriate. In this study we assessed the impact of DTF on health related quality of life (HRQoL). Methods: This was a cross-sectional study done in patients with DTF. The study participants were asked to fill the EORTC QLQ-C30, GAD-7 and PHQ- 9 q uestionnaires to assess HRQoL, anxiety and depression . Outcomes were also compared with healthy controls. Results: 204 subjects (102 DTF patients and 102 healthy controls) were recruited. Study parameters have been summarized in Table. Appendicular skeleton (limbs + girdle) was most commonly involved in 59 % patients and abdominal wall or mesentery was involved in 22.5 %. Patients have received median of 2 lines of therapy. 54 % patients were currently on sorafenib and 41 % were under active surveillance. Mean global health status in DTF patient 65.58 ± 22.64, was significantly lower than healthy controls. Similarly, DTF patients scored low on all functional scales except cognitive functioning. Symptom scale showed significantly higher symptom burden of fatigue, pain, insomnia and financial difficulties. Anxiety & depression was observed in 39.22 % and 50 % of DTF patients respectively. DTF patients had higher rates of mild, moderate and severe anxiety and depression compared to healthy controls. No difference was observed based on site of disease. Conclusions: DTF patients have significant symptom burden, poor functioning, and heightened anxiety and depression. Patient reported outcomes should be routinely used to assess treatment efficacy in DTF patients.[Table: see text]

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

“…Pain, unable to lean on tumour site, fatigue and insomnia were the most prevalent symptoms in our study population. These physical symptoms are consistent with those observed in previous studies in DTF patients and highlight the functional burden DTF patients can experience 9–11,29,30 . Interventions targeting physical symptoms could improve HRQoL of DTF patients, but this requires a careful look at different subgroups with the same degree of symptom burden to know what to intervene on.…”

“…These physical symptoms are consistent with those observed in previous studies in DTF patients and highlight the functional burden DTF patients can experience. 9 , 10 , 11 , 29 , 30 Interventions targeting physical symptoms could improve HRQoL of DTF patients, but this requires a careful look at different subgroups with the same degree of symptom burden to know what to intervene on. To the best of our knowledge, this is the first study that identified relatively distinct subgroups of patients based on their level of symptom burden in a heterogeneous population of DTF patients using LCCA.…”

Desmoid Tumors: A Comprehensive Review

“…The HRQOL burden of patients with DT has been assessed using a number of non–DT-specific instruments such as the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire–Core Module (EORTC QLQ-C30), Generalized Anxiety Disorder scale-7 (GAD-7), Patient Health Questionnaire-9 (PHQ-9), Edmonton Symptom Assessment System-revised (ESAS-r), Distress Assessment and Response Tool (DART), and Child Health Questionnaire (Table 1 ) [ 4 , 39 , 46 , 47 ].…”

“…Thus, it is important that patients be evaluated by a multidisciplinary team with expertise and experience in sarcoma to optimize management. Given the limited mortality associated with DT, treatment goals should not solely focus on clinical markers, such as progression-free survival, but also consider patient-relevant endpoints, such as a reduction in DT-specific symptom burden (e.g., pain) and its impact on patients’ lives, improvement in functioning with daily activities, and overall QOL [ 4 , 40 , 41 , 46 , 47 , 51 ].…”

GOunder/Desmoid Tumor Research Foundation DEsmoid Symptom/Impact Scale (GODDESS©): psychometric properties and clinically meaningful thresholds as assessed in the Phase 3 DeFi randomized controlled clinical trial

“…Treatment of DT requires multidisciplinary team collaboration to create an overall treatment plan with the goal of improving both clinical markers, such as progression-free survival and objective response, as well as patient-relevant outcomes such as desmoid-specific symptoms (e.g., pain), functioning, and overall HRQoL [ 9 – 11 ]. Possible treatment options include systemic therapies, locoregional therapies (e.g., surgery), and/or active surveillance [ 12 ].…”