Table 1.
Author | MKI | N | Detection Method (Tissue) | ORR, % (n; 95% CI) | PFS (Range) | OS (Range) | Grade 3–4 TRAE, % |
---|---|---|---|---|---|---|---|
Retrospective study | |||||||
Gautschi et al.201716 | Cabozantinib | 21 | FISH, PCR, NGS | 37 (7; 16.3–61.5) | 3.6 (1.3–7.0) | 4.9 (1.9–14.3) | Nr |
Vandetanib | 11 | 18 (2; 2.3–51.8) | 2.9 (1.0–6.4) | 10.2 (2.4–NR) | |||
Sunitinib | 10 | 22 (2; 2.8–60.0) | 2.2 (0.7–5.0) | 6.8 (1.1– NR) | |||
Prospective studies—phase 2 | |||||||
Drilon et al.201617 | Cabozantinib | 26 | FISH, NGS | 28 (7; 12–49) | 5.5 (3.8– 8.4) | 9.9 (8.1–NR) | 69 |
Lee et al.201731 | Vandetanib | 18 | FISH, PCR, NGS | 18 (3; Nr) | 4.5 | 11.6 | 28 |
Yoh et al.201732 | Vandetanib | 19 | FISH, PCR | 47 (9; 28–77) | 4.7 (2.8– 8.5) | 11.1 (9.4–NR) | >58 |
Hida et al.201934 | Lenvatinib | 25 | NGS | 16 (4; 4.5–36.1) | 7.3 (3.6– 10.2) | NR | 92 |
PROSPECTIVE STUDIES – Phase 1b | |||||||
Drilon et al.201936 | RXDX-105 | 31 | NGS | 19 (6; 8–38) | Nr | Nr | Nr |
CI, confidence interval; FISH, fluorescence in situ hybridization; MKI, multikinase inhibitor; NGS, next-generation sequencing; NR, not reached; Nr, not reported; ORR, objective response rate; OS, overall survival; PCR, polymerase chain reaction; PFS, progression-free survival; TRAE, treatment-related adverse event.