Background/Problem:
Vorasidenib is an oral brain-penetrant inhibitor of mutant isocitrate dehydrogenase 1 and 2 (IDH1 and 2) enzymes. Vorasidenib has shown to significantly prolong progression–free survival and delay the need for subsequent interventions in treatment-naive patients with IDH–mutant grade 2 gliomas in the pivotal phase III INDIGO trial. Vorasidenib is known to cause deranged liver function which can be dose limiting.
Aim/Goal:
We determined the incidence of liver function (LFT) derangement in real world patients receiving Vorasidenib and evaluated the feasibility of a nurse led model of toxicity monitoring.
Methods/Action:
A single centre retrospective review of patients receiving Vorasidenib between July 2024 and April 2026 through the special access program. Patients were managed via a nurse led telephone model of monthly blood test monitoring and 3 monthly medical review aligned with MRI restaging. Data was collected from electronic medical records. Baseline characteristics and co-morbidities were recorded
Results/Evaluation:
Seven patients were included. Median treatment duration was 394 days (range 220–485). All patients (100%) developed transaminase elevation; two (29%) experienced grade 2 toxicity and one (14%) grade 3 toxicity requiring temporary treatment interruption and dose reduction. Median time to initial LFT elevation was 35 days. Four patients (57%) remained on full-dose therapy and two (29%) continued at a reduced dose. One patient (14%) discontinued treatment due to disease progression; no patients permanently ceased treatment because of hepatotoxicity. Most toxicity episodes were managed independently by the nurse through protocol-driven monitoring and patient communication, reducing unscheduled medical appointments and unplanned hospital admissions.
Discussion:
A nurse-led toxicity monitoring model enabled timely identification and management of vorasidenib-related hepatotoxicity allowing patients to remain on treatment longer. This model appears safe, efficient, and may improve service capacity for patients receiving long-term oral targeted therapies.