Clinical and Molecular Response to Vorasidenib in Post-Transplant Patient with IDH2-mutant Intrahepatic Cholangiocarcinoma
An FDA-approved glioma drug achieved a durable response in an aggressive liver cancer, suggesting genetic targeting may work across different cancer types.
A Johns Hopkins case report of vorasidenib (IDH1/2 inhibitor, FDA-approved for IDH-mutant glioma) achieving durable RECIST partial response and ctDNA reduction in an IDH2-mutant intrahepatic cholangiocarcinoma patient post-liver-transplant, a population with very limited options. This ctDNA-monitored case is hypothesis-generating for IDH-targeted therapy expansion in cholangiocarcinoma and immunosuppressed oncology patients.
What the study was
- Study design
- Case report
- Population
- Single post-transplant patient with IDH2-mutant intrahepatic cholangiocarcinoma, Johns Hopkins
- Sample size
- 1
- Category
- Treatment Innovation
- Maturity
- Exploratory
- Journal
- The Oncologist
Why it surfaced
Novel IDH2-directed therapy in rare post-transplant CCA setting with ctDNA endpoint; hypothesis-generating. Scored 4 (case report cap with rare disease exception for IDH2-mutant CCA).
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