Table 1.
Analysed treatment | Setting | Primary EP | PFS control | PFS gain | PFS HR | OS control | OS gain | OS HR | Adjustment/ Remark | MCBS | MCBS- FT |
Lanreotide versus placebo (CLARINET) Caplin et al, NEJM8 |
Ki-67<10% GI or unknown origin (non-functioning) |
PFS | 18 months | +32% at 2 year | 0.47 (0.30–0.73) |
No improvement in QOL, downgrade 1 point | − | 2 | |||
Octreotide versus placebo (PROMID) Rinke et al, JCO9 |
Midgut, unknown (non-functioning and functioning) |
TTP | 6 months | 8.3 months | 0.34 (0.20–0.59) |
No improvement in QOL, downgrade 1 point | − | 2 | |||
Everolimus versus placebo (RADIANT-4) Yao et al, Lancet10 |
Progressive disease lung or GI (non-functioning) |
PFS | 3.9 months | 7.1 months | 0.48 (0.35–0.67) |
− | 3 | ||||
Everolimus versus placebo (RADIANT-3) Yao et al, NEJM11 Yao et al, JCO12 |
Progressive disease pancreatic NET | PFS | 4.6 months | 6.4 months | 0.35 (0.27–0.45) |
37.7m | 6.3m | Non-significant | − | 3 | |
Octreotide ± everolimus (RADIANT-2) Pavel et al, Lancet13 |
Progressive disease lung, GI, unknown (functioning) |
PFS | 11.3 months | 5.1 months | Non-significant | − | NA | ||||
Sunitinib versus placebo Raymond et al, NEJM14 Faivre et al, JCO15 |
Progressive pancreatic NET | PFS | 5.5 months | 5.9 months | 0.42 (0.26–0.66) |
29.1m | 9.5m | Non-significant | No improvement in QOL, downgrade 1 point | − | 2 |
EP, endpoint; ESMO, European Society for Medical Oncology; FT, field testing; GI, gastrointestinal; MCBS, Magnitude of Clinical Benefit Score; NA, not applicable; NET, neuroendocrine tumour; OS, overall survival; PFS, progression-free survival; QOL, quality of life; TTP, time to progression.