Abstract

305 Background: VHL disease is an inherited condition characterized by the formation of benign and malignant tumors in multiple organs, and RCC affects up to 40-70% of patients with VHL disease. However, real-world evidence on HRU and direct healthcare costs related to VHL-RCC is lacking. This study sought to address the gap in the literature by evaluating HRU and healthcare costs associated with the disease. Methods: Patients with VHL disease were identified from the Optum Clinformatics claims data using an algorithm based on combinations of manifestations of the disease. Among the patients identified, those with at least 1 diagnosis of RCC were further selected. The index date was defined as the date of first observed RCC diagnosis in the data, and the study period extends up to 5 years after index date or the end of continuous enrollment, whichever was earlier. A minimum continuous enrollment of 12 months prior to and 3 months after the index date was required. All-cause and RCC-related HRU and healthcare costs were evaluated among eligible patients with VHL-RCC. HRU was expressed as events per person-month and cost was expressed as average monthly cost. HRU and healthcare costs related to other VHL disease-related tumors were also assessed, including central nervous system (CNS) hemangioblastomas (Hb), retinal Hb, and pancreatic neuroendocrine tumors (pNETs). Results: 160 patients with VHL-RCC were included in the analyses. Mean age of the patients was 51.5 years and 89 (56%) were male. During the study period, patients with VHL-RCC incurred 0.10 hospitalizations (0.52 inpatient days), 1.49 outpatient visits, 0.09 emergency department (ED) visits, and 0.17 other medical visits per person-month on average. This translated to a monthly all-cause healthcare cost of $4,276, which included $2,222 inpatient, $1,318 outpatient, $188 ED, $63 other medical visits, and $485 pharmacy costs. $1,627 out of the all-cause cost per month was RCC-related, which was mainly driven by inpatient costs for RCC ($1,184/month). There were also notable costs associated with other tumor types: $2,159/month for CNS Hb, $601/month for retinal Hb, and $3,306/month for pNETs on average. Among patients with VHL-RCC who received surgical procedures for different tumors, the average hospitalization or outpatient visit costs were $28,356 for nephrectomies, $70,515 for CNS Hb surgeries, $2,887 for laser therapy of retinal Hb, and $81,825 for pNET surgeries. Conclusions: VHL-RCC is associated with significant HRU and healthcare costs due to the needs of managing RCC as well as tumors in other organs caused by VHL disease. This highlights the multi-disciplinary nature of VHL disease and the need for novel effective therapies to prevent or delay the recurrence of VHL-related tumors in order to mitigate the economic burden related to the disease.

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