Table 2.
Single-arm trials in sarcoma patients.
| Reference | Drug | Primaries | Histological subtypes | e-PD | n | Chemo naive, n (%) | 1 prior line, n (%) | ORR | PFR-3 | PFR-6 | Median PFS (months) | Median OS (months) |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Mackay et al. Gynecol Oncol 2012 (10) | aflibercept | Uterine (advanced) | Leiomyosarcoma | NM | 41 | 16 (39%) | 18 (44%) | 0 | ? | 17% | ? | 18.1 |
| Savage et al. Sarcoma 2016 (11) | angiotensin | All (advanced) | all | NM | 20 | 0 | ? | 0 | 45% | ? | 2.7 | 10.2 |
| Chi et al. CCR 2018 (12) | Anlotinib | Soft tissue (advanced) | ASPS | NM | 13 | ? | ? | 6 (46%) | 77% | 77% | 21.0 | Not reached |
| Chi et al. CCR 2018 (12) | Anlotinib | Sott tissue (advanced) | Clear cell sarcoma | NM | 7 | ? | ? | 1 (14%) | 54% | 54% | 11.0 | 16.00 |
| Chi et al. CCR 2018 (12) | Anlotinib | Soft tissue (advanced) | Fibrosarcoma | NM | 18 | ? | ? | 2 (11%) | 81% | 44% | 5.6 | 12.0 |
| Chi et al. CCR 2018 (12) | Anlotinib | Soft tissue (advanced) | Leiomyosarcoma | NM | 26 | ? | ? | 2 (8%) | 75% | 69% | 11.0 | 15.0 |
| Chi et al. CCR 2018 (12) | Anlotinib | Soft tissue (advanced) | Liposarcoma | NM | 13 | ? | ? | 1 (8%) | 63% | 53% | 5.6 | 13.0 |
| Chi et al. CCR 2018 (12) | Anlotinib | Soft tissue (advanced) | Other histologies | NM | 23 | ? | ? | 0 (0%) | 44% | 24% | 2.8 | 8.8 |
| Chi et al. CCR 2018 (12) | Anlotinib | Soft tissue (advanced) | Synovial sarcoma | NM | 47 | ? | ? | 8 (17%) | 75% | 53% | 7.7 | 12.0 |
| Stachiotti et al. EJC 2019 (13) | Axitinib | Soft tissue (advanced) | SFT | Yes | 17 | 9 (53%) | ? | 1/9 (11%) | ? | ? | 9.4 | 25.3 |
| Wilky et al. Lancet Oncol 2019 (14) | Axitinib + Pembrolizumab | Soft tissue (advanced) | ASPS | Yes | 12 | ? | ? | 6/11 (55%) | 72% | 38% | 12.4 | Not reached |
| Wilky et al. Lancet Oncol 2019 (14) | Axitinib + Pembrolizumab | Soft tissue (advanced) | Non-ASPS | Yes | 21 | ? | ? | 2/21 (10%) | 62% | ? | 3.0 | 13.1 |
| Agulnik et al. Ann Oncol 2013 (15) | Bevacizumab | Soft tissue (advanced) | angiosarcoma | NM | 23 | ? | ? | 2/23 (9%) | ? | ? | 3 | 13.2 |
| Agulnik et al. Ann Oncol 2013 (15) | Bevacizumab | Soft tissue (advanced) | EHE | NM | 7 | ? | ? | 2/7 (29%) | ? | ? | 9.8 | 35.5 |
| D’Amado et al. JCO 2005 (16 ) | Doxorubicin + bevacizumab | Soft tissue (advanced) | Leiomyosarcoma | NM | 17 | 11 (65%) | 6 (35%) | 2 (12%) | ? | ? | ? | 16.0 |
| Dickson et al. Sarcoma 2015 (17) | Bevacizumab + Gemcitabine + Docetaxel | Soft tissue (advanced) | All | NM | 35 | 29 (83%) | 17/35 (49%) | 76% | 65% | 7.5 | 28.8 | |
| Monga et al. Cancers 2018 (18) | Bevacizumab + Gemcitabine + Docetaxel + Valproic Acid | Soft tissue (advanced) | All | ? | 46 | 12 (26%) | 15 (33%) | 7/41 (17%) | 5.7 | 12.9 | ||
| Verschraegen et al. Ann Oncol 2012 (19) | Bevacizumab + Gemcitabine + Docetaxel | Soft tissue (neoadjuvant) | All | NM | 15 | 15 (100%) | 0 | 6/15 (40%) | ? | ? | ? | 2y-OS rate : 69% |
| Verschraegen et al. Ann Oncol 2012 (19) | Bevacizumab + Gemcitabine + Docetaxel | Soft tissue (advanced) | All | NM | 20 | 20 (100%) | 0 | 5/20 (25%) | ? | ? | 5.0 | 11.0 |
| Kim et al. Oncologist 2019 (7) | Pazopanib | Soft tissue (advanced) | ASPS | NM | 6 | 4 (67%) | 1 (17%) | 1/6 (17%) | 100% | 50% | 5.5 | Not reached |
| Martin-Broto et al. Lancet Oncol 2019 (20) | Pazopanib | Soft tissue (advanced) | STF (malignant or dedifferentiated) | Yes | 36 | 24 (67%) | 3 (8%) | 2/35 (6%) | 5.6 | Not reached | ||
| Pautier et al. EJC 2020 (21) | Pazopanib + gemcitabine | Uterine and soft tissue (advanced) | Leiomyosarcoma | NM | 105 | 0 (0%) | 105 (100%) | 24/105 (23%) | 6.5 | 24.3 | ||
| Samuels et al. Cancer 2017 (22) | Pazopanib | Soft tissue (advanced) | Liposarcoma (intermediate or high-grade) | NM | 41 | 7 (17%) | 10 (24%) | 1/41 (2%) | 68% | 39% | 4.4 | 12.6 |
| Sleijfer JCO 2009 (23) | Pazopanib | Soft tissue (advanced) | Liposarcoma | Yes | 19 | 1 (5%) | 0 (0%) | 26% | 2.6 | 6.6 | ||
| Sleijfer JCO 2009 (23) | Pazopanib | Soft tissue (advanced) | Leiomyosarcoma | Yes | 42 | 1 (2%) | 1 (2%) | 44% | 3.0 | 11.8 | ||
| Sleijfer JCO 2009 (23) | Pazopanib | Soft tissue (advanced) | Synovialosarcoma | Yes | 38 | 0 (0%) | 5 (13%) | 49% | 5.3 | 10.3 | ||
| Sleijfer JCO 2009 (23) | Pazopanib | Soft tissue (advanced) | Other histologies | Yes | 43 | 0 (0%) | 3 (7%) | 39% | 3.0 | 9.9 | ||
| Stacchiotti Lancet Oncol 2019 (24) | Pazopanib | Soft tissue (advanced) | extraskeletal myxoid chondrosarcoma | Yes | 26 | 21 (81%) | 2 (8%) | 4/26 (15%) | 19.0 | Not reached | ||
| Subbiah CCR 2017 (25) | Pazopanib + trametinib | Soft tissue (advanced) | All | NM | 25 | 0 (0%) | 2/25 (8%) | 2.3 | 9.0 | |||
| Bompas Anna Oncol 2015 (26) | Sorafenib | Bone (advanced) | Chordoma | NM | 27 | 15 (56%) | ? | 2/27 (7%) | 85% | Not reached | Not reached | |
| Chevreau et al. Cancer 2013 (27) | Sorafenib | Soft tissue | EHE | Yes | 15 | 10 (67%) | ? | 2/15 (13%) | 38% | 6.0 | Not reached | |
| D’Adamo et al. The Oncologist 2019 (28) | Sorafenib + DTIC | Soft tissue (advanced) | LMS+MPNST+SS | NM | 37 | 16 (43%) | 11 (30%) | 5/37 (14%) | 51% | 25% | 3.3 | 13.2 |
| Garcia Del Muro Invest New Dugs 2018 (29) | Sorafenib + Ifosfamide | Soft tissue (advanced) | All | NM | 35 | 3 (9%) | 6/35 (17%) | 66% | 37% | 4.8 | 16.2 | |
| Grignani et al. Ann Oncol 2012 (30) | Sorafenib | Bone (advanced) | Osteosarcoma | NM | 35 | 0 (0%) | 3/35 (9%) | 17% | 4.0 | 7.0 | ||
| Grignani et al. Lancet Oncol 2015 ( 30 ) | Sorafenib + everolimus | Bone (advanced) | Osteosarcoma | NM | 38 | 0 (0%) | 2/38 (5%) | 45% | 5 | 11 | ||
| Von Mehren et al. Cancer 2012 (31) | Sorafenib | Soft tissue (advanced) |
All | NM | 37 | 18 (49%) | 0/37 (0%) | 3.0 | 17.0 | |||
| Pacey et al. Invest New Drugs 2011 (32) | Sorafenib | Soft tissue (advanced) | All | NM | 21 | 0 (0%) | 7 (33%) | 3/21 (14%) | 14% | 0% | ||
| Raut et al. PLOSOne 2012 (33) | Sorafenib | Soft tissue (advanced) | All | NM | 15 | 1 (7%) | 0/15 (0%) | |||||
| Ray-Coquard et al. Oncologist 2012 (34) | Sorafenib | Soft tissue (advanced) |
angiosarcoma | NM | 41 | 11 (27%) | 16 (39%) | 6/41 (15%) | 12% | |||
|
Santoro et al. Ann Oncol
2013 (35) |
Sorafenib | Soft tissue (advanced) | All | NM | 101 | 0 (0%) | 40 (40%) | 11/76 (14%) | 35% | 4.2 | 11.9 | |
| Valentin et al. Invest New Drugs 2013 (36) | Sorafenib | Soft tissue | SFT | NM | 5 | 3 (60%) | 2 (40%) | 0 | 19.7 | |||
|
Gounder et al.
Clin Cancer res 2011 (37) |
Sorafenib | Soft tissue (advanced) | Desmoid tumors | NM | 26 | 11 (42%) | 6/24 (25%) | |||||
| George et al. J Clin Oncol 2009 (38) | Sunitinib | Soft tissue (advanced) | All | NM | 48 | 1/48 (2%) | 14.5% | |||||
| Jo et al. Invest New Drugs 2014 (39) | Sunitinib | Soft tissue (advanced) | Desmoid tumors | NM | 19 | 11 (58%) | 4 (21%) | 5 (26%) | ||||
| Agulnik et al. Ann Oncol 2017 (15) | Tivozanib | Soft tissue (advanced) | All | NM | 58 | 0 (0%) | 20 (34%) | 2/58 (3%) | 3.5 | 12.2 | ||
| Cohen et al. Pediatr Blood Cancer 2019 (40) | Cediranib | Soft tissue | ASPS | NM | 7 | 3 (43%) | 0/7 (0%) | |||||
| Kummar et al. JCO 2013 (41) | Cediranib | Soft tissue | ASPS | NM | 46 | 18 (39%) | 18 (39%) | 15/43 (35%) | 36/43 (84%) |
e-PD, disease progression at entry in the study; ORR, objective response rate according to RECIST; PFR, progression-free survival rate; PFS: progression-free survival; OS, overall survival; NM, not mandatory; ASPS, alveolar soft tissue part sarcoma; SFT, solitary fibrous tumor; EHE, epithelioid hemangioendothelioma; LMS, leiomyosarcoma; MPNST, malignant peripheral nerve sheath tumor; SS, synovial sarcoma.