Pembrolizumab for Patients With Refractory or Relapsed Thymic Epithelial Tumor: An Open-Label Phase II Trial
Phase 2N = 33NCT02607631
- Population
- Patients with histologically confirmed thymic epithelial tumour whose disease progressed after at least one line of platinum-based chemotherapy; 33 enrolled (26 thymic carcinoma, 7 thymoma).
- Design
- Open-label phase II trial. Pembrolizumab 200 mg intravenously every 3 weeks until tumour progression or unacceptable toxicity. Patients with active autoimmune disease requiring systemic treatment within the past year, or a documented history of clinically severe autoimmune disease, were excluded. Primary objective: response rate, assessed every 9 weeks by investigators. Thymoma and thymic carcinoma were reported separately.
- Primary endpoint
- ORR
- Arms
- Thymic carcinoma (experimental, n = 26): Pembrolizumab 200 mg intravenously every 3 weeks
- Thymoma (experimental, n = 7): Pembrolizumab 200 mg intravenously every 3 weeks
- People
- Cho J
Show the numbers
| Endpoint | Analysis | Thymic carcinoma / Thymoma | Control | Effect |
|---|---|---|---|---|
| ORREnrolled patients by histology (26 thymic carcinoma, 7 thymoma); investigator assessment | primaryCho 2019 | 19.2% (8.5–37.9%) | — | Partial responses in 5 of 26 patients with thymic carcinoma and 2 of 7 with thymoma; the abstract mentions no complete response. Stable disease in 14 patients (53.8%) with thymic carcinoma and in 5 with thymoma (the abstract prints 71.6%, whereas 5 of 7 is 71.4%). Most common adverse events of any grade: dyspnoea 11 (33.3%), chest wall pain 10 (30.3%), anorexia 7 (21.2%) and fatigue 7 (21.2%). |
| PFSEnrolled patients by histology (26 thymic carcinoma, 7 thymoma) | primaryCho 2019 | median 6.1 months | — | The abstract gives one value for both histologic groups and no confidence interval. |
| Grade 3 or worse immune-related adverse eventsEnrolled patients by histology (26 thymic carcinoma, 7 thymoma) | primaryCho 2019 | 15.4% | — | Grade 3 or worse immune-related adverse events in 4 of 26 patients with thymic carcinoma and 5 of 7 with thymoma. Events named (percentages of all 33 patients): hepatitis 4 (12.1%), myocarditis 3 (9.1%), myasthenia gravis 2 (6.1%), thyroiditis 1 (3.0%), ANCA-associated rapidly progressive glomerulonephritis 1 (3.0%), colitis 1 (3.0%) and subacute myoclonus 1 (3.0%). |
| Endpoint | Analysis | Thymic carcinoma / Thymoma | Control | Effect |
|---|---|---|---|---|
| ORREnrolled patients by histology (26 thymic carcinoma, 7 thymoma); investigator assessment | primaryCho 2019 | 19.2% (8.5–37.9%) | — | Partial responses in 5 of 26 patients with thymic carcinoma and 2 of 7 with thymoma; the abstract mentions no complete response. Stable disease in 14 patients (53.8%) with thymic carcinoma and in 5 with thymoma (the abstract prints 71.6%, whereas 5 of 7 is 71.4%). Most common adverse events of any grade: dyspnoea 11 (33.3%), chest wall pain 10 (30.3%), anorexia 7 (21.2%) and fatigue 7 (21.2%). |
| PFSEnrolled patients by histology (26 thymic carcinoma, 7 thymoma) | primaryCho 2019 | median 6.1 months | — | The abstract gives one value for both histologic groups and no confidence interval. |
| Grade 3 or worse immune-related adverse eventsEnrolled patients by histology (26 thymic carcinoma, 7 thymoma) | primaryCho 2019 | 15.4% | — | Grade 3 or worse immune-related adverse events in 4 of 26 patients with thymic carcinoma and 5 of 7 with thymoma. Events named (percentages of all 33 patients): hepatitis 4 (12.1%), myocarditis 3 (9.1%), myasthenia gravis 2 (6.1%), thyroiditis 1 (3.0%), ANCA-associated rapidly progressive glomerulonephritis 1 (3.0%), colitis 1 (3.0%) and subacute myoclonus 1 (3.0%). |
- Thymic carcinoma (experimental)
- Thymoma (experimental)
Median-only curves assume a constant hazard (exponential) and fit plateaus, as seen with some immunotherapies, poorly.
Data table (modelled values)
| Months | Thymic carcinoma | Thymoma |
|---|---|---|
| 0 | 100.0% | 100.0% |
| 4 | 63.5% | 63.5% |
| 8 | 40.3% | 40.3% |
| 11 | 28.7% | 28.7% |
| 15 | 18.2% | 18.2% |