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. 2015 Jan;7(1):22–38. doi: 10.1177/1758834014551936

Table 1.

Initial trials of sorafenib in thyroid carcinoma: phase II and retrospective studies.

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.