Real-World Characterization and Treatment Patterns of Patients with Desmoid Tumors at an Academic Center in the United States

美国某学术中心纤维瘤患者的真实世界特征及治疗模式

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Abstract

Real-world studies on desmoid tumors (DT) are limited due to its low incidence. Therefore, we aimed to characterize the patient journey in DTs from diagnosis to treatment at an academic center in the United States. A retrospective cohort study was conducted at the University of Utah Health network. Eligible patients were diagnosed with DTs between January 1, 2011, and July 31, 2023, were ≥18 years old at diagnosis, and had ≥2 DT-related encounters. Patient and tumor characteristics, reports of symptoms, and treatment patterns were abstracted from electronic health records. Healthcare resource utilization was quantified using administrative claims. Patients who potentially had a misdiagnosis ≤2 years before DT diagnosis were identified based on billing codes of conditions commonly diagnosed instead of DTs. Among 148 eligible patients, 59.5% had documentation of ≥1 DT-related symptom during the follow-up period. Pain was the most common reported symptom (57.4%). 79.7% of patients received ≥1 line of active treatment; some received up to 10 lines of therapy. Healthcare resource utilization was higher during periods of symptomatic disease and among patients with potential misdiagnosis (29.1%) in the year before DT diagnosis. Although surgery was the most common treatment, its use decreased across the study period with a corresponding expansion of systemic treatment modalities used as first-line treatment. In conclusion, increased healthcare resource utilization associated with misdiagnosis highlights the need for timely and accurate DT diagnosis. The varied treatment approaches and multiple lines of therapy suggest the need for optimal therapeutic agents. Surgery rates have decreased, aligning with evolving treatment guidelines. SIGNIFICANCE: DT remains an understudied disease area despite its high morbidity. Due to limited data on treatment patterns and outcomes, particularly in the United States, gaps in the management of DTs are not well elucidated. Therefore, we aimed to describe the patient journey in DTs within our health system.

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