Related Experiment Video
Updated: May 18, 2026
Intramucosal Inoculation of Squamous Cell Carcinoma Cells in Mice for Tumor Immune Profiling and Treatment Response Assessment
Published on: April 22, 2019
Immune Checkpoint Inhibitors in Unresectable or Metastatic Thymic Epithelial Tumors--A Real-World Assessment of
Guilherme Sacchi de Camargo Correia1, Reema Tawfiq2, Ruqin Chen2
1Division of Hematology/Oncology, Mayo Clinic, Jacksonville, FL; Georgia Cancer Center at Augusta University, Augusta, GA.
Abstract:
Thymic epithelial tumors (TET) are rare, with limited prospective data on their management. While studied in the second-line setting and beyond, real-world data on immune checkpoint inhibitors (ICIs) outcomes are lacking. We assessed the real-world efficacy and safety of ICIs in TET, and investigated the correlation between immune-related adverse events (irAEs) and clinical outcomes. We retrospectively reviewed patients with unresectable or metastatic TET treated with ICIs at 5 Mayo Clinic sites. Baseline characteristics, outcomes, and toxicity data were collected. We calculated the overall response rate (ORR), median progression-free survival (mPFS), median overall survival (mOS), and assessed their correlation with irAEs. Thirty patients were included (28 with thymic carcinoma, 2 with thymoma). Pembrolizumab was used in 29 patients, and durvalumab in one. The ORR was 23.3%. The mPFS was 10.12 months and did not differ by irAEs incidence (hazard ratio (HR) 0.99, 95% confidence interval (CI), 0.39-2.54). 40.0% of patients had irAEs, which were predominantly grade 2 (61.5%), and most commonly thyroiditis (26.7%). The mOS was 81.3 months, numerically longer in those with irAEs versus those without (81.3 vs. 58.6 months; HR 0.51, 95% CI, 0.13-1.97). The ORR was similar to data published in the literature, although mPFS and mOS were numerically longer. The incidence of irAEs was higher but less severe than in prior data. irAEs did not significantly correlate with outcomes, despite a trend toward prolonged mOS. Further studies are needed to identify biomarkers, optimize both patient selection and toxicity management, and improve TET outcomes.
Insights
Real-world data show immune checkpoint inhibitors (ICIs) offer a 23.3% response rate in thymic epithelial tumors (TET). Immune-related adverse events (irAEs) were common but did not significantly impact survival outcomes.
Area of Science:
- Oncology
- Immunotherapy
- Rare Cancers
Background:
- Thymic epithelial tumors (TET) are rare malignancies with limited real-world data on advanced treatment strategies.
- Immune checkpoint inhibitors (ICIs) show promise, but their efficacy and safety in TET require further investigation.
Purpose of the Study:
- To evaluate the real-world effectiveness and safety of ICIs in patients with unresectable or metastatic TET.
- To explore the relationship between immune-related adverse events (irAEs) and clinical outcomes in this patient population.
Main Methods:
- Retrospective review of 30 patients with unresectable or metastatic TET treated with ICIs across five Mayo Clinic sites.
- Data collected included baseline characteristics, treatment response (ORR), progression-free survival (mPFS), overall survival (mOS), and toxicity (irAEs).
Main Results:
- The overall response rate (ORR) was 23.3%, with a median progression-free survival (mPFS) of 10.12 months.
- 40% of patients experienced irAEs, predominantly grade 2, with thyroiditis being the most common.
- Median overall survival (mOS) was 81.3 months, numerically longer in patients with irAEs, though this did not reach statistical significance.
Conclusions:
- Real-world ICI treatment for TET demonstrated an ORR comparable to literature, with numerically longer mPFS and mOS.
- While irAEs were frequent, they were generally less severe than previously reported and did not significantly correlate with clinical outcomes.
- Further research is needed to identify predictive biomarkers, optimize patient selection, and manage toxicity to improve TET outcomes.
