Table 1.
Trials of Immune Checkpoint inhibitors in advanced Soft-tissue Sarcomas.
| Trial | Design | Molecule tested | Population/Histotypes | Overall Response Rate | Median progression free survival | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall | LMS | LPS | UPS | Synovial sarcoma | ASPS | Overall | LMS | LPS | UPS | Synovial sarcoma | ASPS | ||||
| Trials for multiple histotypes | |||||||||||||||
| SARC028 (38) | Non-randomized, Phase 2 trial | Pembrolizumab 200mg flat dose q3w | Four cohorts of 10 patients in each histotype: LMS, DDLPS, UPS, synovial sarcoma |
17.5% | 0% | 20% | 40% | 0% | 15w | 25w | 30w | 7w | |||
| SARC028 expansion cohorts (39) | Non-randomized, Phase 2 trial | Pembrolizumab 200mg flat dose q3w | Two cohorts: 39 DDLPS and 40 UPS | 10% | 23% | 2M | 3M | ||||||||
| Alliance A091401 (40) | Multicentre, open-label, non comparative, Phase 2 trial | Nivolumab 3mg/kg q2w | 42 STS | 5% | 1.7M | ||||||||||
| Nivolumab 3mg/kg + ipilimumab 1mg/kg q3w for 4 cycles followed by nivolumab 3mg/kg q2w |
41 STS | 16% | 4.1M | ||||||||||||
| Alliance A091401 expansion cohorts (41) | Multicenter, open-label, non comparative, phase 2 trial | Nivolumab 3mg/kg q2w | 15 DDLPS & 13 UPS | 6.7% | 7.7% | 4.6M | 1.5M | ||||||||
| Nivolumab 3mg/kg + ipilimumab 1mg/kg q3w for 4 cycles followed by nivolumab 3mg/kg q2w |
14 DDLPS & 14 UPS | 14.3% | 28.6% | 5.5M | 2.7M | ||||||||||
| Somaiah et al. (36) | Non-randomized, Phase 2 trial | Durvalumab 1500mg + tremelimumab 75mg q4w for 4 cycles followed by durvalumab alone |
57 soft-tissue sarcomas: 6 LPS, 5 UPS, 5 synovial sarcoma, 10 ASPS and others |
14.3% | 2.8M | 2M | 1.8M | 7.46M | 34.23M | ||||||
| Trials for specific histotypes | |||||||||||||||
| Maki et al. (42) | Non-randomized, Two-stage, Phase 2 trial (terminated early due to lack of efficacy) |
Ipilimumab 3mg/kg q3w | 6 synovial sarcomas | 0% | 1.85 months | ||||||||||
| Ben-Ami et al. (43) | Non-randomized, Two-stage, Phase 2 trial (terminated early due to lack of efficacy) |
Nivolumab 3mg/kg q2w | 12 uterine LMS | 0% | 1.8 months | ||||||||||
| Blay et al. (44) | Non-randomized, Phase 2 trial | Pembrolizumab 200mg q3w | Rare sarcomas (incidence<0.2/100.000): 24 chordoma, 14 ASPS, 5 DSRCT, 6 SMARCA4-malignant rhabdoid tumors & 32 others |
15% | 35.7% | 7.9 months | |||||||||
| OSCAR (45) | Single arm, Phase 2 trial | Nivolumab 240mg q2w | 11 clear cell sarcomas and 14 ASPS | 4% | 7.1% | 4.9 months | 6 months | ||||||||
| Gxplore-005 (46) | Single arm, Phase 2 trial | Geptanolimab 3mg/kg q2w | 37 ASPS | 37.8% | 6.9 months | ||||||||||
ASPS, Alveolar Soft Part Sarcoma; DDLPS, Dedifferentiated Liposarcoma; DSRCT, desmoplastic small round cell tumor; LMS, Leiomyosarcoma; LPS, Liposarcoma; UPS, Undifferentiated Pleomorphic Sarcoma.