Case report on vorasidenib treatment for Grade 2 oligodendroglioma and astrocytoma in Chinese patients, highlighting the safety profile and adverse events.
Vorasidenib (VOR), a brain-penetrant dual inhibitor of mutant IDH1/2 (mIDH1/2), is US.FDA-approved for Grade 2 mIDH1/2 diffuse glioma but has not yet been approved in Asia. Clinical evidence in Asian populations remains limited. Leveraging the temporary importation policy of Hainan’s Boao Pilot Zone, we report our single-center experience—the earliest real-world VOR cases in Asia. Case #1: 56-yo female predominantly Grade 2 (with a small portion of Grade 3) oligodendroglioma, IDH1 R132H mutation and 1p/19q co-deletion, no prior radio-chemotherapy. Case #2: 35-yo female Grade 2 oligodendroglioma, IDH1 R132H mutation and 1p/19q codeletion, prior chemotherapy. Case #3: 41-yo female Grade 2 astrocytoma, IDH1 mutation, prior radio-chemotherapy. Case #4: 40-yo male Grade 2 astrocytoma, IDH2 R172S mutation, prior radio-chemotherapy. Case #5: 55-yo male Grade 2 astrocytoma, IDH1 mutation, prior radio-chemotherapy. Case #6: 50-yo male Grade 2 oligodendroglioma, IDH1 R132H mutation and 1p/19q codeletion, no prior radio-chemotherapy. Case #7: 37-yo male Grade 2 astrocytoma, IDH1 R132H mutation, no prior radio-chemotherapy. Four patients (Cases 1, 2, 4, and 5) received VOR at 40 mg once daily for 6 months. Case 6 received VOR for 3 months, and Case 7 for 1 month. One patient (14.3%) had Grade 2 AST/ALT elevation; others reported Grade 1 or less liver function or GI events(4 cases), or no adverse events observed_2 cases). Slight tumor volume reduction was observed in Case 5 following 6 months of VOR treatment. The remaining six patients exhibited stable disease throughout their treatment duration. These 7 cases show a safety profile consistent with the Phase 3 study and potential efficacy. Follow-up is ongoing to assess safety and effectiveness. As the earliest real-world experience in Asia, these findings may provide evidence to support clinical decision-making for the regional patient population.
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Chen et al. (2025) studied this question.
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