First-line toripalimab in combination with platinum-doublet chemotherapy for advanced B3 thymoma and thymic carcinoma: an open-label prospective phase Ⅱ trial.
7 affiliations
- 1Department of Medical Oncology, National Cancer Center/National Clinical Research Center for Cancer/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
- 2Department of Medical Oncology, National Cancer Center/National Clinical Research Center for Cancer/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China; Department of Hepatobiliary Oncology, Beijing Tsinghua Changgung Hospital, School of Clinical Medicine, Tsinghua Medicine, Tsinghua University, Beijing, China.
- 3Department of Medical Oncology, Beijing Chao Yang District San Huan Cancer Hospital, Beijing, China.
- 4General Department, Beijing Chaoyang Huanxing Cancer Hospital, Beijing, China.
- 5Geneseeq Research Institute, Nanjing Geneseeq Technology Inc., Nanjing, Jiangsu, China.
- 6Department of Medical Oncology, National Cancer Center/National Clinical Research Center for Cancer/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China. Electronic address: [email protected].
- 7Department of Medical Oncology, National Cancer Center/National Clinical Research Center for Cancer/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China. Electronic address: [email protected].
Abstract
Background
Several studies have demonstrated the efficacy of programmed cell death protein 1/programmed death-ligand 1 (PD-L1) inhibitors in patients with thymic epithelial tumors (TETs). Previous trials suggest that the efficacy of immunotherapy in TETs is associated with specific features of the tumor immune microenvironment.Patients and methods
Patients received a combination of paclitaxel, carboplatin, and toripalimab every 3 weeks for up to six cycles, followed by toripalimab maintenance every 4 weeks until disease progression or other discontinuation criteria. The primary endpoint was progression-free survival (PFS). Immunohistochemistry and next-generation sequencing were used to assess the association between various biomarkers and efficacy.Results
Between November 2020 and January 2024, 24 patients were enrolled in the study. The median PFS was 19.3 months [95% confidence interval (CI) 12.8-25.8]. The objective response rate was 37.5% (95% CI 18.8%-59.4%) and the disease control rate was 100% (95% CI 85.8%-100%). Grade 3-4 treatment-related adverse events (TRAEs) occurred in 12 (50.0%) patients. Common grade 3-4 TRAEs included decreased neutrophil, white blood cell, and platelet counts. Immune-related adverse events (irAEs) were identified in 11 (45.8%) patients; grade ≥3 irAEs were reported in 1 (4.2%) patient. Patients with higher PD-L1 expression showed a trend toward longer PFS. Further analysis revealed a specific infiltration pattern of CD8+ T cells, M1/M2 macrophages, and CD56bright natural killer cells in the tumor immune microenvironment and mutations in the nuclear factor κB pathway were associated with better clinical outcomes.Conclusions
Toripalimab combined with carboplatin and paclitaxel is an emerging therapeutic option for patients with thymic carcinoma and B3 thymoma. The overall incidence and severity of adverse events are within the acceptable range. Tumor microenvironment features or mutational signatures are credible prognostic indicators.
Related
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BibTeX
@article{zhu2026firstline,
title = {{First-line toripalimab in combination with platinum-doublet chemotherapy for advanced B3 thymoma and thymic carcinoma: an open-label prospective phase Ⅱ trial.}},
author = {K Zhu and H Zhu and H Shi and J Lu and Y Feng and M Wang and D Chen and X Ji and P Xing and N Bai and S Wang and Y Liu and X Hu},
journal = {ESMO open},
year = {2026},
month = oct,
doi = {10.1016/j.esmoop.2026.108366},
url = {https://europepmc.org/article/MED/42854640},
note = {CC BY-NC-ND},
}
RIS
TY - JOUR
TI - First-line toripalimab in combination with platinum-doublet chemotherapy for advanced B3 thymoma and thymic carcinoma: an open-label prospective phase Ⅱ trial.
AU - K Zhu
AU - H Zhu
AU - H Shi
AU - J Lu
AU - Y Feng
AU - M Wang
AU - D Chen
AU - X Ji
AU - P Xing
AU - N Bai
AU - S Wang
AU - Y Liu
AU - X Hu
PY - 2026
DA - 2026/10/09
JO - ESMO open
DO - 10.1016/j.esmoop.2026.108366
UR - https://europepmc.org/article/MED/42854640
AB - Background Several studies have demonstrated the efficacy of programmed cell death protein 1/programmed death-ligand 1 (PD-L1) inhibitors in patients with thymic epithelial tumors (TETs). Previous trials suggest that the efficacy of immunotherapy in TETs is associated with specific features of the tumor immune microenvironment.Patients and methods Patients received a combination of paclitaxel, carboplatin, and toripalimab every 3 weeks for up to six cycles, followed by toripalimab maintenance every 4 weeks until disease progression or other discontinuation criteria. The primary endpoint was progression-free survival (PFS). Immunohistochemistry and next-generation sequencing were used to assess the association between various biomarkers and efficacy.Results Between November 2020 and January 2024, 24 patients were enrolled in the study. The median PFS was 19.3 months [95% confidence interval (CI) 12.8-25.8]. The objective response rate was 37.5% (95% CI 18.8%-59.4%) and the disease control rate was 100% (95% CI 85.8%-100%). Grade 3-4 treatment-related adverse events (TRAEs) occurred in 12 (50.0%) patients. Common grade 3-4 TRAEs included decreased neutrophil, white blood cell, and platelet counts. Immune-related adverse events (irAEs) were identified in 11 (45.8%) patients; grade ≥3 irAEs were reported in 1 (4.2%) patient. Patients with higher PD-L1 expression showed a trend toward longer PFS. Further analysis revealed a specific infiltration pattern of CD8+ T cells, M1/M2 macrophages, and CD56bright natural killer cells in the tumor immune microenvironment and mutations in the nuclear factor κB pathway were associated with better clinical outcomes.Conclusions Toripalimab combined with carboplatin and paclitaxel is an emerging therapeutic option for patients with thymic carcinoma and B3 thymoma. The overall incidence and severity of adverse events are within the acceptable range. Tumor microenvironment features or mutational signatures are credible prognostic indicators.
SN - 2059-7029
ER -