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Case Report: Potential Predictive Value of MMR/MSI Status and PD-1 Expression in Immunotherapy for Urothelial
Yu-Ting Ma1, Hong-Lan Yang2, Li Yan3
1Department of Pathology, Shandong Provincial Hospital Affiliated to Shandong First Medical University, Jinan, China.
Abstract:
Immune checkpoint inhibitors (ICIs) have shown encouraging outcomes against Lynch syndrome (LS)-associated colorectal cancer (CRC) and endometrial cancer with mismatch repair deficient/microsatellite instability-high (dMMR/MSI-H). However, there is as yet no clarity on the safety and efficacy of immunotherapy combined with chemotherapy in LS-associated urothelial carcinoma (UC). Here, we report a patient with recurrent and metastatic LS-associated UC who achieved sustained response to programmed death protein 1 (PD-1) inhibitor combined with chemotherapy over 31 months, during which the side effects of immunotherapy could be controlled and managed. Our findings indicate that the dMMR/MSI status and PD-1 expression in UC may have potential predictive value for the response to PD-1-targeted immunotherapy. Our case supports the inclusion of such combination and/or monotherapy for UC in clinical studies and using dMMR/MSI status and PD-1 expression as potential predictive biomarkers for assessment of the therapeutic response.
Insights
Immune checkpoint inhibitors combined with chemotherapy show promise for Lynch syndrome-associated urothelial carcinoma. Mismatch repair deficient/microsatellite instability-high status may predict response to PD-1 inhibitors.
Area of Science:
- Oncology
- Immunotherapy
- Genitourinary Cancers
Background:
- Immune checkpoint inhibitors (ICIs) are effective in Lynch syndrome (LS)-associated colorectal and endometrial cancers with mismatch repair deficiency (dMMR) or microsatellite instability-high (MSI-H) status.
- The safety and efficacy of combining immunotherapy with chemotherapy for LS-associated urothelial carcinoma (UC) remain unclear.
Observation:
- A patient with recurrent and metastatic LS-associated UC experienced a sustained response to a programmed death protein 1 (PD-1) inhibitor plus chemotherapy for over 31 months.
- The side effects associated with the immunotherapy regimen were manageable.
Findings:
- The patient's dMMR/MSI status and PD-1 expression in UC showed potential as predictive biomarkers for response to PD-1-targeted immunotherapy.
- This case demonstrates the feasibility and sustained efficacy of combination therapy in a challenging UC subtype.
Implications:
- The findings support the investigation of PD-1 inhibitor combination or monotherapy for urothelial carcinoma in clinical studies.
- dMMR/MSI status and PD-1 expression are proposed as valuable predictive biomarkers for assessing therapeutic response in UC patients.
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