Table 1.
Study design | Inclusion criteria | Number of patients | BRAF mutation | Response rate | PFS (months) | OS (months) | |
---|---|---|---|---|---|---|---|
Gupta-Abramson et al. [2008] | Phase II prospective | PTC (18)
FTC (9) MTC (1) PD/ATC (2) |
30 | – | PR = 7 (23%)
SD = 16 (53%) |
21 | – |
Kloos et al. [2009] | Phase II prospective | Arm A:
PTC (19) Arm B: PTC (22) FTC (11) ATC (4) |
46 | PTC = 17/22
nPTC = 0/6 |
PRPTC = 6 (14%)
PRnPTC = 0 (0%) SDPTC = 25 (61%) SDnPTC = 10 (67%) |
10–16 (nPTC = 4.5) | 23–37 (nPTC = 24.2) |
Cabanillas et al. [2010] | Retrospective | PTC (8)
FTC (7) |
15 | PTC = 4/7 | 12 (80%)
PR = 3 (20%) SD = 9 (60%) |
19 | 67% at 2 years |
Lam et al. [2010] | Phase II
prospective |
sMTC (16)
hMTC (5) |
21 | RET mutation:
sMTC = 10/12 hMTC = 5/5 |
PR = 1 + 1 (9.5%)
SD = 8 + 1 (43%) |
17.9 | – |
Ahmed et al. [2011] | Phase II prospective | PTC (8)
FTC (9) PDTC (2) MTC (15) |
34 | DTC = 1/3 | PR = 5 (15%)
SD = 25 (73%) |
71% at 2 years | 79% at 2 years |
Schneider et al. [2012] | Phase II prospective | DTC | 31 | BRAF: 10/32
K/N-RAS: 3/9 PIK3CA: 2/6 |
PR=8 (31%)
SD=11 (42%) |
18 | 34.5 |
Capdevila et al. [2012] | Retrospective | PTC (7)
FTC (9) MTC (15) ATC (3) |
34 | – | PRDTC = 3 (19%)
PRMTC = 7 (47%) PRATC = 1 (33%) SDDTC = 8 (50%) SDMTC = 6 (40%) SDATC = 0 (0%) |
10.5
DTC = 13.3 MTC = 10.5 ATC = 4.4 |
23.6
DTC = 23.6 MTC = NR ATC = 5 |
Savvides et al. [2013] | Phase II prospective | ATC | 20 | – | PR = 2 (10%)
SD = 5 (25%) |
1.9 | 3.9 |
ATC, anaplastic thyroid carcinoma; DTC, differentiated thyroid carcinoma; FTC, follicular thyroid carcinoma; hMTC, hereditary medullary thyroid carcinoma; MTC, medullary thyroid carcinoma; nPTC, nonpapillary thyroid carcinoma; OS, overall survival; PDTC, poorly differentiated thyroid carcinoma; PFS, progression-free survival; PR, partial response; PTC, papillary thyroid carcinoma; SD, stable disease; sMTC, sporadic medullary thyroid carcinoma.