Table 8.
FT of the ESMO-MCBS for the treatment of GIST and soft tissue sarcomas at the Medical University of Vienna
| Analysed treatment | Setting | Primary EP | PFS control | PFS gain | PFS HR | OS control | OS gain | OS HR | Adjustment/ remark | MCBS | MCBS- FT |
| Sunitinib versus placebo* Demetri et al 53 |
Second-line advanced GIST after imatinib | TTP | 6.4 weeks | 16.9 weeks | 0.33 (0.23–0.47) |
3 | − | ||||
| Regorafenib versus Placebo (GRID)* Demetri et al 54 |
third-line advanced GIST after imatinib and sunitinib | PFS | 0.9 months | 3.7 months | 0.27 (0.19–0.39) |
3 | − | ||||
| Doxorubicin ± ifosfamide (EORTC 62012) Judson et al 55 |
Previously untreated soft tissue sarcoma | OS | 13 months | 1.5 months | Non-significant | Results do no support intensified treatment | − | NA | |||
| High-dose doxorubicin + ifosfamide versus doxorubicin Maurel et al 56 |
Previously untreated soft tissue sarcoma | PFS | 26 weeks | Non-significant | Results do no support intensified treatment | − | NA | ||||
| Pegylated liposomal doxorubicin versus doxorubicin Judson et al 57 |
Naïve or pretreated advanced soft tissue sarcoma | RR | 9% | 1% | Less toxicity, upgrade 1–2 points | − | 1–3 | ||||
| Doxorubicin +/- olaratumab Tap et al 58 |
Previously untreated soft tissue sarcoma | PFS | 4.1 months | 2.5 months | 0.67 (0.44–1.02) |
14.7 months | 11.8 months | 0.46 (0.30–0.71) |
Improvement in survival -> form 2a | − | 4 |
| Pazopanib versus placebo (PALETTE)* van der Graaf et al 59 |
Previously treated soft tissue sarcoma | PFS | 1.6 months | 3.0 months | 0.31 (0.24–0.4) |
3 | − | ||||
| Gemcitabine + dacarbazine versus gemcitabine García-Del-Muro et al 60 |
Previously treated soft tissue sarcoma | PFS 3 months | 2 months | 2.2 months | 0.58 (0.39–0.86) |
8.2 months | 8.6 months | 0.56 (0.36–0.9) |
Improvement in survival -> form 2a | − | 4 |
| Trabectedin q21 versus trabectedin q28 d1+8+15 Demetri et al 61 |
Previously treated liposarcoma, leiomyosarcoma | TTP | 2.3 months | 1.4 months | 0.73 (0.55–0.97) |
Data support use of trabectedin | − | 2 | |||
| Trabectedin versus dacarbazine Demetri et al 62 |
Previously treated liposarcoma, leiomyosarcoma | OS | 1.5 months | 2.7 months | 0.55 (0.44–0.70) |
12.4 months | 0.5 months | Non-significant | PFS as second endpoint improved | − | NA |
| Eribulin versus dacarbazine Schöffski et al 63 |
Previously treated liposarcoma, leiomyosarcoma | OS | – | – | – | 11.5 months | 2 months | 0.77 (0.62–0.95) |
– | 2 |
*Aadapted according to Cherny et al. 1
†Unclear toxicity data.
EP, endpoint; ESMO, European Society for Medical Oncology; FT, field testing; GIST, gastrointestinal stromal tumours; MCBS, Magnitude of Clinical Benefit Score; OS, overall survival; PFS, progression-free survival; TTP, time to progression.