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Table 1 Panoramic view of clinical trials and retrospective studies with novel drugs for anaplastic thyroid cancer

From: Recent advances in the management of anaplastic thyroid cancer

Pharmacodynamic Property Tested Drug(s) First Author Study Design ATC patients N°
(out of total)
Prior ATC cohort therapies (%) ATC Staging (%) ORR (%) DCR (%) Median OS, months
CTX XRT Surgery IVA IVB IVC
mTKI Lenvatinib Tahara Phase II 17 (out of 51) 7 (41) 9 (53) 14 (82) 4 (24) 5 (29) 6 (35) 4/17 (24) 16/17 (94) 10.6
Iwasaki Retrospective 23 (out of 23) 0 (0) 0 (0) 10 (43) 0 (0) 0 (0) 23 (100) 4/23 (17)A 10/23 (44)A 5.5B
Iyer Retrospective 16 (out of 16) 9 (69) 7 (44) 8 (50) 0 (0) 4 (25) 12 (75) 3/10 (30) 7/10 (70) 3.9
Sorafenib Savvides Phase II 20 (out of 20) 20 (100) 18 (90) 18 (90) 0 (0) 0 (0) 20 (100) 2/20 (10) 7/20 (35) 3.9
Ito Phase II 10 (out of 18) 6 (60) 7 (70) 7 (70) 1 (10) 0 (0) 8 (80) 0/10 (0) 4/10 (40) 5.0
Sunitinib Ravaud Phase II 4 (out of 71) NA NA NA 0 (0) 0 (0) 4 (100) 0/4 (0) 1/4 (25) NA
PP-compounds
CLM3/24/29
- - - - - - - - - - - -
Vandetanib - - - - - - - - - - - -
Axitinib Cohen Phase II 2 (out of 60) NA NA NA NA NA NA 1/2 (50) 1/2 (50) NA
Pazopanib Bible Phase II 15 (out of 15) 11 (73) 12 (80) NA 0 (0) 1 (7) 14 (93) 0/15 (0) 0/15 (0) 3.7
Imatinib Ha Phase II 11 (out of 11) 9 (82) 7 (64) 0 (0) 5 (45) 6 (55) 2/8 (25)C 6/8 (75)C NAD
Single target EGFR Gefitinib Pennell Phase II 5 (out of 27) 3 (60) NA NA 0 (0) 5 (100) 0/5 (0) 1/5 (20) NA
BRAF Vemurafenib Hyman Phase II 7 (out of 122) 7 (100) 6 (86) NA NA NA NAE 2/7 (28) 2/7 (28) NA
Dabrafenib +
Trametinib
Subbiah Phase II 16 (out of 100) 6 (38) 13 (81) 14 (88) 0 (0) 16 (100) 11/16 (69) 14/16 (88) NA
Iyer Retrospective 16 (out of 16) 9 (69) 7 (44) 8 (50) 0 (0) 4 (25) 12 (75) 3/6 (50) 5/6 (83) 9.3
mTOR Everolimus Lim Phase II 6 (out of 40) 2 (33) 0 (0) 6 (100) 0/6 (0) 0/6 (0) NA
Schneider Phase II 7 (out of 35) NA NA NA NA NA NA 0/7 (0) 0/7 (0) 2.8
Hanna Phase II 7 (out of 50) 3 (43) 4 (57) 5 (71) 0 (0) 0 (0) 7 (100) 1/7 (14) 3/7 (43) 4.6
PPARγ TZDs - - - - - - - - - - - -
VEGFR-2 Cyclic amide CLM94 - - - - - - - - - - - -
Vascular disrupting Fosbretabulin Mooney Phase II 26 (out of 26) 13 (50) 17 (65) 19 (73) 7 (27) 19 (73) 0/26 (0) 7/26 (27) 4.7
Immunotherapy
(anti PD-1/PD-L1)
Spartalizumab Capdevila Phase II 38 (out of 42)F 25 (60) 30 (71) 28 (67) NA NA NA 8/42 (19) 13/42 (31) 5.9
Combination therapy Sorafenib +
Temsirolimus
Sherman Phase II 2 (out of 36) NA NA NA NA NA NA 1/2 (50) 1/2 (50) NA
Efatutazone + Paclitaxel Smallridge Phase I 15 (out of 15)G 4 (27) 8 (53) 11 (73) 0 (0) 4 (27) 11 (73) 0/7 (0) vs.
1/6 (17)
4/7 (57) vs.
4/6 (67)
3.3 vs.
4.6
Fosbretabulin +
CP
Sosa Phase II/III 80 (out of 80) 19 (35) 21 (38) 30 (55) 1 (2) 4 (7) 49 (89) 11/55 (20) 22/55 (40) 5.2
  1. Abbreviations: CTX Chemotherapy (including both traditional and novel drugs); XRT Radiotherapy; ORR Overall Response Rate; DCR Disease Control Rate; OS Overall Survival; mTKI multiple Tirosine-Kinase Inhibitor; PP Pyrazolo [3,4-d]pyrimidine; EGFR Epidermal Growth Factor Receptor; mTOR mammalian Target Of Rapamycin; PPARγ Peroxisome Proliferator-Activated Receptor Gamma; TZDs Thiazolidinediones; VEGFR Vascular Endothelial Growth Factor Receptor; PD-1 Programmed cell Death protein 1; PD-L1 Programmed death-ligand 1; CP Paclitaxel followed by Carboplatin.
  2. NOTES
  3. ABOR was not evaluable in 6 patients
  4. BMedian OS in patients treated surgically vs. patients treated with Lenvatinib only was 8.8 months vs. 4.3 months, respectively
  5. COnly 8 of 11 enrolled patients were evaluable for BOR
  6. DThe rate of 6-month OS was 45%EAt least 6 patients (86%) presented at distance metastasis
  7. FAll data reported refer to the full cohort of patients enrolled, including 4 whose diagnosis of ATC was not histologically confirmed. Most patients had metastatic disease, with lungs and lymph nodes as the most common sites
  8. GORR, DCR and OS refer to Paclitaxel + Efatutazone 0.15 mg vs. Paclitaxel + Efatutazone 0.3 mg cohorts, respectively (two patients adimistered with Efatutazone 0.5 mg only incurred disease progression during the run-in phase)