Table 4 Summary results of studies that deployed novel therapeutic modalities in patients with distant metastases from thyroid carcinomas

Kloos et al., 2009

Total 56 (44.6%): Treatment group 19 (42%); Control group 37 (45.9%)

-

Papillary (73%), Follicular (4%), Hurthle cell (16%), Anaplastic (7%)

BM (21%)

Evaluate the activity of sorafenib in metastatic thyroid carcinoma

Treatment group; sorafenib 400 mg PO bd

Lung (96), lymph (94%), other (14%)

Partial response in 15% of patients (7.5 months median duration). Median progression-free survival was 15 months

Cabanillas et al., 2010

15 (60%)

61 (38–83)

PWD (53.3%), FWD (33.3%), Hurthle (13.3%)

BM (26.7%)

Evaluate the activity of sorafenib and sunitinib in progressive and radioactive resistant DTC

Sorafenib 200–400 mg PO bd 86.7%, sunitinib 50 mg PO od 13.3%

Lymph (73.3%), lung (66.7%), pleura (13.3%)

SD 9 (60%), PR 3 (20%), PD 3 (20%). 2 yr survival 67%

Orita et al., 2011

Total 50 (72%): Treatment group 22 (68.2%); Control 28 (75%)

59 (32–77)

PWD (52%), FWD (48%)

SM (40%), BM (100%)

Evaluate the efficacy of ZA for treatment of symptomatic BM in DTC

Treatment group; ZA 4 mg IV/month (100%), Surgery (18%), ERT (77%), RAI (50%)

Others (60%)

Treatment group/control SRE-free 3 yr survival: 86%/50%, mortality 54%

Wells et al., 2012

Total 331 (42.6%): Treatment group 231 (42%); Control group 100 (44%)

-

Advanced MTC (100%)

Treatment group BM (34%), control group BM (40%)

Evaluate the efficacy of vandetanib in advanced or metastatic MTC

Treatment group; vandetanib 300 mg PO

Hepatic (46.5%), lymph nodes (40.8%), respiratory (38.1%), neck (10.0%)

Significant prolongation of PFS with vandetanib compared with placebo. PD 37%, mortality 15%