Abstract 148P
Background
Desmoid tumors (aggressive fibromatosis) can negatively affect patients’ lives and overall quality of life due to the associated symptoms and complications. This analysis describes patient-reported symptom severity and impact of desmoid tumors on participants’ lives.
Methods
This is an analysis of the global, survey-based Desmoid Tumor Research Foundation (DTRF) Natural History Study using data collected from Sept 2017 to Aug 2023. Participants and/or their caregivers reported a desmoid tumor diagnosis, symptoms, and the impact on their lives. Symptom severity and impact were evaluated using a desmoid tumor-specific measure: the GOunder/DTRF Desmoid Symptom Scale (GODDESS© DTSS) and Impact Scale (GODDESS DTIS), where higher scores indicate worse symptoms or more severe impact (Qual Life Res 2023;32:2861). Mean scores for participants with a current tumor versus those without were compared using Analysis of Variance (ANOVA); other comparisons were descriptive.
Results
367 participants completed the GODDESS surveys (median age, 39 years). Participants reported a high symptom burden, with an overall total symptom mean score of 4.1 out of 10. Item scores for pain, fatigue, and impaired mobility were significantly higher (worse) in participants with a current tumor versus those without (Table). Desmoid tumors had a negative impact on the participants’ reported physical, sleep, and emotional domain scores (Table). Participants with abdominal wall desmoid tumors reported numerically higher (worse) symptom scores for pain, fatigue, and emotional items versus participants with intra-abdominal, extra-abdominal, and/or other tumor locations.
Table: 148P
GODDESS DTSS and DTIS domain scores
| Current tumor | |||
| Yes n=247 | No n=106 | p-valuea | |
| DTSS scoresb | |||
| Total symptom score | 4.4 | 3.4 | 0.002 |
| Pain domain (worst feeling of…) | 4.5 | 3.5 | 0.002 |
| Pain | 4.9 | 3.8 | 0.003 |
| Dull pain | 4.1 | 3.3 | 0.012 |
| Shooting pain | 4.5 | 3.3 | 0.001 |
| Fatigue | 5.0 | 4.1 | 0.011 |
| Extra-abdominal symptom domain (worst…) | 4.0 | 3.1 | 0.010 |
| Swelling around tumor(s) | 3.6 | 2.5 | 0.004 |
| Difficulty moving near the tumor(s) | 4.8 | 3.5 | 0.001 |
| Abdominal symptoms domain (worst feeling of…) c | 4.0 | 3.6 | 0.409 |
| Abdominal pain | 4.8 | 4.3 | 0.412 |
| DTIS domain scores | |||
| Physical function d | 1.7 | 1.4 | 0.005 |
| Sleep d | 1.8 | 1.5 | 0.026 |
| Emotion b | 4.4 | 3.9 | 0.125 |
aANOVA, p<0.05 b11-point Numerical Rating Scale cCompleted by participants with relevant tumors d5-point Likert scale
Conclusions
The pain and impaired physical functioning and emotional well-being reported by participants can have a negative impact on their lives. Effective care of patients with desmoid tumors depends on understanding the real-world burden and impact of these tumors.
Clinical trial identification
Editorial acknowledgement
Writing and editing support was provided by Rebekka Harding-Smith and Jessica Warren of IQVIA with funding from SpringWorks Therapeutics, Inc.
Legal entity responsible for the study
Desmoid Tumor Research Foundation.
Funding
SpringWorks Therapeutics.
Disclosure
K.A. Mercier: Financial Interests, Institutional, Research Grant: SpringWorks Therapeutics, Inc.; Non-Financial Interests, Personal, Principal Investigator: Desmoid Tumor Research Foundation. L. Hernandez: Financial Interests, Institutional, Research Grant: SpringWorks Therapeutics, Inc.; Non-Financial Interests, Personal, Other, Executive Director: Desmoid Tumor Research Foundation. A. Lucas: Financial Interests, Institutional, Research Grant: SpringWorks Therapeutics, Inc.; Non-Financial Interests, Personal, Other, Staff Associate: Desmoid Tumor Research Foundation. T. Bell, B. Tumminello, S. Zhou: Financial Interests, Personal, Full or part-time Employment: SpringWorks Therapeutics, Inc..