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‹ Sat · 11 Jul 2026
Novel or significantly improved treatment

Long-Term Outcomes and Surgical Conversion After Immunotherapy in Microsatellite Instability-H Biliary Tract Cancers.

Rare biliary tract cancers with specific genetic features respond exceptionally well to immunotherapy, with 73% surviving two years.

In a multicenter retrospective cohort of MSI-H biliary tract cancer patients treated with immune checkpoint inhibitors, 2-year overall survival was 73%, with 23.1% of patients achieving surgical conversion and 50% of resected patients showing pathological complete response. These findings support upfront dMMR/MSI-H testing in biliary tract cancers and establish that this rare molecular subset derives exceptional benefit from immunotherapy.

What the study was

Study design
multicenter retrospective cohort study
Population
MSI-H biliary tract cancer patients, multicenter US cohort (Mayo/Michigan)
Category
Treatment Innovation
Maturity
Validated
Journal
JCO precision oncology

Why it surfaced

Establishes durable benefit and surgical conversion from ICI in rare MSI-H BTC (Mayo/Michigan multicenter); directly actionable for upfront dMMR/MSI-H testing policy in biliary tract cancers.

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