An Assessment of Financial Burden and its Association with Desmoid Tumor Specific Symptoms and Impacts in Adult Desmoid Tumor Patients: Desmoid Tumor Research Foundation Natural History Study
Author(s)
Sophia Zhou, MS1, Amanda Lucas, BS2, Lynne Hernandez, BA2, Yucheng Chu, MS3, Timothy Bell, MHA1, Ana B Oton, MD1, Kelly Mercier, PhD4.
1SpringWorks Therapeutics, Inc., Stamford, CT, USA, 2Desmoid Tumor Research Foundation, Philadelphia, PA, USA, 3Sumptuous Data Sciences, Monmouth Junction, NJ, USA, 4Desmoid Tumor Research Foundation; Duke University, Philadelphia, PA, USA.
1SpringWorks Therapeutics, Inc., Stamford, CT, USA, 2Desmoid Tumor Research Foundation, Philadelphia, PA, USA, 3Sumptuous Data Sciences, Monmouth Junction, NJ, USA, 4Desmoid Tumor Research Foundation; Duke University, Philadelphia, PA, USA.
Presentation Documents
OBJECTIVES: Evaluate adult patient-reported financial burden of desmoid tumors (DT) and its association with DT-specific pain or function via the global, survey-based Desmoid Tumor Research Foundation (DTRF) Natural History Study using data collected from September 2017 to July 2024.
METHODS: Participants responded to the cancer-specific financial questionnaire of Patient Reported Outcomes (PRO) for Fighting Financial Toxicity (PROFFIT, J Cancer Policy. 2023 Dec; 38:100440), and the GOunder/DTRF DEsmoid Tumor Symptom Scale (GODDESS© DTSS) and Impact Scale (GODDESS© DTIS) PRO tool for DT-specific symptoms and impact on their lives. Median financial toxicity (FT) scores (higher scores indicate worse financial burden) were compared using a Mann-Whitney test for presence vs absence of a current DT or a prior DT treatment, and a Kruskal-Wallis test for DT location subgroups. The association of PROFFIT with DT-specific pain or function was assessed by linear regression.
RESULTS: In total, 107 participants (median age: 44 years) completed the PROFFIT survey. Median FT score was 24 (scale: 0-100) for DT, comparable to malignancies like breast cancer, thoracic tumor, gynecologic tumor, gastrointestinal colorectal or non-colorectal cancer (median range: 14-36, AnnResOncol 2022;2(2):94). No significant differences were observed in FT scores across tumor locations (abdominal wall, intra-abdominal, joint/extremity or other, median range: 19-33) or between participants with vs without a prior treatment (19 vs 26). Participants with a current tumor (n=58) had a significantly higher FT score (median: 29 vs 14, P=.047) than those without (n=23). Worsening of FT was significantly associated with worsening in the DTSS pain domain (P=.01) and DTIS domains of sleep, physical or emotional functioning (P<.0001).
CONCLUSIONS: DT can impose financial burden associated with DT-specific pain and function limitations similar to that for malignant cancers. Understanding the impact of patient reported financial toxicity on health outcomes could help improve the management of patients with DT.
METHODS: Participants responded to the cancer-specific financial questionnaire of Patient Reported Outcomes (PRO) for Fighting Financial Toxicity (PROFFIT, J Cancer Policy. 2023 Dec; 38:100440), and the GOunder/DTRF DEsmoid Tumor Symptom Scale (GODDESS© DTSS) and Impact Scale (GODDESS© DTIS) PRO tool for DT-specific symptoms and impact on their lives. Median financial toxicity (FT) scores (higher scores indicate worse financial burden) were compared using a Mann-Whitney test for presence vs absence of a current DT or a prior DT treatment, and a Kruskal-Wallis test for DT location subgroups. The association of PROFFIT with DT-specific pain or function was assessed by linear regression.
RESULTS: In total, 107 participants (median age: 44 years) completed the PROFFIT survey. Median FT score was 24 (scale: 0-100) for DT, comparable to malignancies like breast cancer, thoracic tumor, gynecologic tumor, gastrointestinal colorectal or non-colorectal cancer (median range: 14-36, AnnResOncol 2022;2(2):94). No significant differences were observed in FT scores across tumor locations (abdominal wall, intra-abdominal, joint/extremity or other, median range: 19-33) or between participants with vs without a prior treatment (19 vs 26). Participants with a current tumor (n=58) had a significantly higher FT score (median: 29 vs 14, P=.047) than those without (n=23). Worsening of FT was significantly associated with worsening in the DTSS pain domain (P=.01) and DTIS domains of sleep, physical or emotional functioning (P<.0001).
CONCLUSIONS: DT can impose financial burden associated with DT-specific pain and function limitations similar to that for malignant cancers. Understanding the impact of patient reported financial toxicity on health outcomes could help improve the management of patients with DT.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
SA13
Topic
Study Approaches
Topic Subcategory
Registries
Disease
SDC: Oncology, SDC: Rare & Orphan Diseases