TABLE I.
Selected completed immunotherapy studies in soft-tissue sarcoma (STS)
| Reference | Agent | Phase | Pts (n) | Indication | Response rate | Survival |
|---|---|---|---|---|---|---|
| Checkpoint inhibitors | ||||||
| Maki et al., 201319 | Ipilimumab | I | 6 | Advanced SyS | 0 of 6 | mOS: 8.75 months |
| Tawbi et al., 201716 | Pembrolizumab | II | Selected STSs and bone sarcomas | 18% in STS, 40% in UPS, 20% in LPS, 10% in SyS | mPFS: 18 weeks; OS: 49 weeks | |
| D’Angelo et al., 201821 | Nivolumab with or without ipilimumab | II | 96 | Metastatic STS | Nivolumab: 5%; Ipilimumab–nivolumab: 16% | mPFS: 4.1 months; OS: 14.3 months |
| Toulmonde et al., 201826 | Pembrolizumab, cyclophosphamide | II | 57 | Advanced STS | Solitary fibrous tumour in 1 patient | NA |
| Wilky et al., 201922 | Axitinib, pembrolizumab | II | ASPS and other STSs | 25%, all STS patients; 50.4%, ASPS patients | 3-Month PFS: 66%; in ASPS patients: 73% | |
| Adoptive cell therapy | ||||||
| Robbins et al., 201127 | Adoptively transferred autologous T cells transduced with a T cell receptor directed against NY-ESO-1 | I | 6 | Metastatic SyS expressing NY-ESO-1 |
4 of 6 | NA |
| Vaccines | ||||||
| Mahvi et al., 200228 | Tumour cells treated with granulocyte macrophage colony–stimulating factor | I | 16 | Melanoma and sarcoma | 1 of 16 | NA |
| Dillman et al., 200429 | Autologous tumour cell-line-derived vaccines | I/II | 23 | Recurrent or metastatic sarcoma | No objective response | 10 Patients lived more than 1 year |
| Kawaguchi et al., 200530 | Vaccination with SYT–SSX junction peptide | I | 6 | Metastatic SyS | 0 of 6 | NA |
| Finkelstein et al., 201231 | Radiotherapy with intratumoural injection of dendritic cells | I/II | 17 | Neoadjuvant treatment in high-risk STS | 9 of 17 | 1-Year PFS: 70.6% |
| Kawaguchi et al., 201232 | SYT–SSX breakpoint peptide vaccines | I/II | 21 | Metastatic SyS | 1 of 21 (stable disease: 6 of 21) | NA |
Pts = patients; SyS = synovial sarcoma; mOS = median overall survival; UPS = undifferentiated pleomorphic sarcoma; LPS = liposarcoma; mPFS = median progression-free survival; OS = overall survival; NA = not applicable; ASPS = alveolar soft-part sarcoma; PFS = progression-free survival.