Real-world monitoring and treatment patterns in von Hippel-Lindau (VHL) disease-associated central nervous system hemangioblastomas (CNS-Hb).

Abstract

2086 Background: VHL disease is an inherited tumor syndrome that causes multiple benign and malignant tumors to develop; CNS-Hb is often the first VHL disease manifestation, occurring in 70-80% of patients with VHL. Patients often need many tumor treatments to manage the disease. This study evaluated disease monitoring and treatment patterns among patients with VHL-CNS-Hb. Methods: Using an algorithm based on VHL manifestations, patients with VHL were identified from Optum’s de-identified Clinformatics Data Mart Database. Patients with a CNS-Hb diagnosis were then selected. Treatment patterns were assessed among patients with VHL after their initial diagnosis of CNS-Hb. Incidence rates (IR) of the following treatments for managing CNS-Hb or other VHL-associated tumors were assessed: surgery for removal of VHL-associated tumors, renal ablation, radiotherapy, targeted therapy for RCC, and retinal laser therapy. Rates for tumor treatments were reported as events per 10-person years. Generalized linear models estimated IRs of analgesic use, VHL disease monitoring procedures, and visits to VHL-related medical specialists in the patient and control cohorts, adjusting for age, sex, the number of outpatient visits at baseline, and health plan type. Results: 220 patients with VHL-CNS-Hb were identified. Reflecting the multifaceted nature of VHL disease, the tumor treatments with the highest unadjusted IRs during the study period among these patients were targeted RCC therapies (2.15 per 10-person years), surgical removal of cerebellar and spinal hemangioblastomas (0.52 per 10-person years), and radiotherapy (0.42 per 10-person years). The tablecontains estimated adjusted IRs and IRRs for the use of select analgesics, monitoring procedures, and medical specialist visits for the VHL-CNS-Hb and comparator cohorts. Conclusions: VHL-CNS-Hb presents a multifaceted burden, involving treatments for various VHL-related tumors. Patients with VHL-CNS-Hb use more analgesics, undergo frequent monitoring, and have increased specialist visits compared with the control group. Successful tumor control holds the potential to reduce morbidity and long-term medical management associated with the disease. [Table: see text]