Characterization of Patients With Desmoid Tumors (DT) and Their Treatment Patterns Within an Academic Health System in the United States
Moderator
Chia Jie Tan, RPh, PhD, University of Utah, Salt Lake City, UT, United States
Speakers
Connor Willis, PharmD, University of Utah, Salt Lake City, UT, United States; Timothy Bell, SpringWorks Therapuetics, Stamford, CT, United States; Brad Tumminello; Sophia Zhou, Cary, NC, United States; Dipam Doshi; Carl V Asche, BA, MBA, MSc, PhD, University of Utah, Salt Lake City, UT, United States; Joseph Biskupiak, MBA, PhD; Diana Brixner, RPh, PhD, University of Utah, Salt Lake City, UT, United States; Anna Chalmers; David Stenehjem
OBJECTIVES: To assess characteristics and treatment patterns of adult patients with DT.
METHODS: A retrospective cohort study was conducted at the University of Utah. Eligible patients had DT diagnosis between 1JAN2011 and 31JUL2023 confirmed by pathology, were aged >18 years when diagnosed, and had >2 DT clinical encounters. Patient and tumor characteristics, clinical journey, and treatment patterns were assessed using electronic health records. Misdiagnoses were identified in the study ≤2 years before DT diagnosis based on billing codes of conditions commonly diagnosed instead of DT. Healthcare resource utilization data in outpatient, emergency, and inpatient settings were quantified using administrative claims.
RESULTS: A total of 148 patients met eligibility criteria. At diagnosis, median age was 36 years (interquartile range [IQR]: 27-46); most patients were female (68.2%); median DT size was 60mm (IQR: 40-92). Abdominal wall was the most common tumor location (27.7%). Misdiagnoses were identified in 29.1% of patients and associated with higher healthcare resource utilization in the year before DT diagnosis than patients with DT without misdiagnosis. Most patients (89.0%) (median follow-up = 27 months) reported >1 episode of active disease (DT-related symptoms, treatment and/or progression) and 79.7% received >1 line of treatment (LOT; range: 1-10), among whom 33.9% received >3 LOT. Multiple treatment modalities were employed, including surgery, radiotherapy, chemotherapy, and targeted therapy. Surgery was the most common modality (52.7%), but surgery at first LOT decreased from 74.2% (46/62) in 2011-2017 to 44.6% (25/56) in 2018-2023.
CONCLUSIONS: The occurrence of misdiagnosis and increased healthcare utilization suggest the need for more timely and accurate DT diagnosis. The wide range of treatment approaches, multiple LOT, and high rate of active disease support developing better therapeutic agents. Patients receiving surgery for DT as first LOT decreased from 2011-2017 to 2018-2023, consistent with surgery generally not recommended as first LOT in guidelines.
METHODS: A retrospective cohort study was conducted at the University of Utah. Eligible patients had DT diagnosis between 1JAN2011 and 31JUL2023 confirmed by pathology, were aged >18 years when diagnosed, and had >2 DT clinical encounters. Patient and tumor characteristics, clinical journey, and treatment patterns were assessed using electronic health records. Misdiagnoses were identified in the study ≤2 years before DT diagnosis based on billing codes of conditions commonly diagnosed instead of DT. Healthcare resource utilization data in outpatient, emergency, and inpatient settings were quantified using administrative claims.
RESULTS: A total of 148 patients met eligibility criteria. At diagnosis, median age was 36 years (interquartile range [IQR]: 27-46); most patients were female (68.2%); median DT size was 60mm (IQR: 40-92). Abdominal wall was the most common tumor location (27.7%). Misdiagnoses were identified in 29.1% of patients and associated with higher healthcare resource utilization in the year before DT diagnosis than patients with DT without misdiagnosis. Most patients (89.0%) (median follow-up = 27 months) reported >1 episode of active disease (DT-related symptoms, treatment and/or progression) and 79.7% received >1 line of treatment (LOT; range: 1-10), among whom 33.9% received >3 LOT. Multiple treatment modalities were employed, including surgery, radiotherapy, chemotherapy, and targeted therapy. Surgery was the most common modality (52.7%), but surgery at first LOT decreased from 74.2% (46/62) in 2011-2017 to 44.6% (25/56) in 2018-2023.
CONCLUSIONS: The occurrence of misdiagnosis and increased healthcare utilization suggest the need for more timely and accurate DT diagnosis. The wide range of treatment approaches, multiple LOT, and high rate of active disease support developing better therapeutic agents. Patients receiving surgery for DT as first LOT decreased from 2011-2017 to 2018-2023, consistent with surgery generally not recommended as first LOT in guidelines.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HSD30
Topic
Health Service Delivery & Process of Care
Disease
SDC: Rare & Orphan Diseases