Patients with von Hippel-Lindau-associated central nervous system hemangioblastoma (VHL-CNS-Hb) have a large economic burden, but there is limited real-world evidence describing their clinical burden in the United States (US). This study compared treatment patterns and monitoring procedures between patients with VHL-CNS-Hb and matched controls in the US.

Patients with VHL-CNS-Hb and matched controls were identified from Optum’s de-identified Clinformatics^® Data Mart Database. The index date was the date of first CNS-Hb diagnosis (VHL-CNS-Hb cohort) or the date of a random medical claim within the patient’s claims (control cohort). Treatment patterns were assessed in the VHL-CNS-Hb cohort using unadjusted incidence rates. Pain management drug use, disease monitoring procedures, and visits to medical specialists were compared between cohorts using unadjusted and adjusted generalized linear models. Clinical trial number: not applicable.

The most common treatment observed in the VHL-CNS-Hb cohort (N = 220) was targeted therapy for renal cell carcinoma (2.34 treatment events per 10 person-years). Pain management drug use, monitoring, and specialist visits peaked during the first/second year post-index. Compared to controls (N = 1100), VHL-CNS-Hb patients had higher rates of pain management drug use (adjusted incidence rate ratio [IRR]: 1.96), monitoring procedures (adjusted IRR: 3.93), and specialist visits (adjusted IRRs: 2.49–25.44; all P ≤ 0.001).

Patients with VHL-CNS-Hb had a substantial, long-term clinical burden that included treatment for multiple VHL manifestations—reflecting the multi-organ nature of the disease—frequent pain management drug use, and high levels of healthcare use for disease monitoring and specialist visits.