Table 2 Clinical studies with targeted therapies in patients with anaplastic thyroid cancer

Imatinib

8

2 (25%)

4 (50%)

6 (36%)

45%

Ha et al., JCO, 2010 [26] Phase II

Sorafenib

20

2 (10%)

5 (25%)

1.9

3.9

30%

Savvides et al., Thyroid, 2013 [27] Phase III

Pazopanib

15

0

0

2.0

3.6

13%

Bible et al., JCEM 2012 [28] Phase II

Everolimus

7

0

3 (43%)

2.2

2.5

0%

Lim et al., Ann Oncol., 2013 [29] Phase II

Everolimus

7

0

0

4.3

Schneider et al. J Clin Endocrinol Metab. 2017 [30] Phase II

Everolimus

7

1 (14%)

1 (14%)

2.2

4.6

Hanna et al. Clin Cancer Res. 2018 [31] Phase II

Fosbretabulin

(CA4P)

3

1 (33%)

CR > 30 mo

0

Dowlati et al., Cancer Res, 2002 [32] Phase II

Fosbretabulin

26

0

7 (27%)

3 (23%)

4.7

34%

Mooney et al., JCO 2006 [33] Phase II

Fosbretabulin/carboplatin/paclitaxel

80

20%

40%

3.3

5.2

26% (1y)

Sosa et al., Thyroid, 2014 [34] Phase III

Axitinib

2

1 (50%)

Cohen et al., JCO 2014 [35] Phase II

Gefitinib

1

1 (100%, > 4 mo)

Fury et al., Cancer Chemother Pharmacol 2007 [36] Phase I

Gefitinib

5

0

1 (20%, > 12 mo)

Pennell et al., Thyroid, 2008 [37] Phase II

Larotrectinib

7

2 (29%)

1 (14%)

Cabanillas et al., Ann Oncol 2020 [38] Phase II

Sunitinib

4

0

1 (25%)

9.8

Ravaud et al. Eur J Cancer 2017 [39] Phase II

Lenvatinib + Pembrolizumab

6

4 (66%)

1 (16%)

16.5

18.5

83%

Dierks et al. Thyroid 2021 [31] retrospective data

Dabrafenib + Trametinib

16

1 (6,2%)

10 (62%)

3 (19%)

62%

Subbiah et al. JCO 2018 [40] Phase II

Pembrolizumab (1 pat Nivolumab)

13

16% ORR

1.9

4.4

38% (1y)

Hatashima Thyroid 2022 [ 41] Case series

Dabrafenib + Trametinib, Vemurafenib

17, 4

6.4

14.5

De la Fouchardière Ann Endocrin 2022 [42] retrospective data