Dados do Resumo

Título

ASSESSMENT OF HEMOGLOBIN AND ERYTHROPOIETIN LEVELS IN PATIENTS WITH VON HIPPEL-LINDAU SYNDROME UNDERGOING BELZUTIFAN TREATMENT

Introdução

Von Hippel-Lindau (VHL) syndrome is caused by pathogenic germline variants in the VHL gene, predisposing individuals to retinal and CNS angiomas, renal cancer, neuroendocrine tumors, and other neoplasms. Recently, the HIF2A inhibitor Belzutifan has shown efficacy in treating all tumors associated with VHL.

Objetivo

This subproject main aim is to prospectively record the hemoglobin and erythropoietin levels of patients with VHL who have a medical indication for treatment with the oral medication Belzutifan, before the start (baseline) and throughout the first 6 months of treatment. It's secundary aim is to evaluate other adverse effects during the first 6 months of treatment, and assess the need for subcutaneous erythropoietin supplementation in patients with anemia.

Métodos

A real-world clinical study on the use of Belzutifan in VHL was recently approved by the institutional ethics committee, and 35 VHL patients with different tumors have been included since October 2023. We have collected baseline (pre-treatment) variables from laboratory tests, such as complete blood counts and plasma erythropoietin (EPO) levels, and we'll followed the results monthly for up to 6 months of treatment. A form containing the adverse effects will be provided for the participants to fill out with the symptoms they experience during the treatment.

Resultados

Preliminary results indicate that all participants developed grade 1-2 anemia, with fatigue and headache being the primary adverse effects (grade 1-2). In five participants with symptomatic anemia or a significant hemoglobin drop in the first 2 months of treatment, subcutaneous (SC) EPO was supplemented efficiently, with prompt recovery.

Conclusões

In conclusion, although guidelines for EPO supplementation are not yet defined, we observed that an initial dose of 4,000 IU per week SC was effective for prompt recovery of acute symptoms and improvement of hemoglobin levels in all cases. Maintenance EPO supplementation is still under discussion in our cohort.

Palavras Chave

von Hippel-Lindau; VHL; Belzutifan