Table 2.
Selected clinical trials that assessed KIT inhibitors in melanoma.
| Study | Phase | N | Subtype (n) | Arms | ORR (%) | DCR (%) | PFS (mo) | OS (mo) |
|---|---|---|---|---|---|---|---|---|
| Kim et al. 2008 [86] | 2 b | 21 | Cutaneous (7) Acral (2) Soft part (1) Unclassified (11) |
Imatinib | 4.8 | 23.8 | 1.4 | 7.5 |
| Guo et al. 2011 [87] | 2 a | 43 | Acral (21) Mucosal (11) Cutanous (9) Unknown (2) |
Imatinib | 23.3 | 53.5 | 3.5 | 14 |
| Carvajal et al. 2011 [88] | 2a | 28 | Mucosal (13) Acral (10) Cutaneous (5) |
Imatinib | Mucosal 23 Acral 38 Cutaneous 0 |
NS | 2.8 | 10.7 |
| Hodi et al. 2013 [89] | 2 a | 24 | Mucosal (17) Acral (6) Cutenous (1) |
Imatinib | 29 | 50 | 3.7 | 12.5 |
| Cho et al. 2012 [90] | 2 a | 11 | Acral (9) Mucosal (2) |
Nilotinib | 22.2 | 77.8 | 2.5 | 7.7 |
| Carvajal et al. 2015 [91] | 2 a,e | 19 | Mucosal (12) Acral (4) Cutaneous (3) |
Nilotinib | Mucosal 27.2 Acral 0 Cutaneous 0 |
Mucosal 63.6 Acral 25 Cutaneous 33.3 |
3.4 f 2.6 g |
14.2 f 4.3 g |
| Lee et al. 2015 [92] | 2 b | 27 | Acral (15) Mucosal (7) Cutaneous (5) |
Nilotinib | Acral 40 Mucosal 0 Cutaneous 0 |
Acral 73.3 Mucosal 28.6 Cutaneous 40 |
NS | NS |
| Guo et al. 2017 [93] | 2 b | 42 | Acral (20) Mucosal (20) Cutaneous (2) |
Nilotinib | Acral 25 Mucosal 25 Cutaneous 50 |
Acral 80 Mucosal 70 Cutaneous 50 |
4.2 | 18 |
| Deylon et al. 2018 [94] | 2 b | 22 | Mucosal (9) Acral (7) Cutaneous (6) |
Nilotinib | Mucosal 33.3 Acral 14.3 Cutaneous 16.6 |
Mucosal 66.6 Acral 71.4 Cutaneous 80 |
6 d | 13.2 d |
| Kalinsky et al. 2016 [95] | 2 b | 25 | Acral (15) Mucosa (10) |
Dasatinib | Acral 33 Mucosal 14 |
50 c | 2.7 | 11.8 |
| Minor et al. 2012 [96] | - b | 6 | Mucosal (NS) Acral (NS) Cutaneous (NS) |
Sunitinib | Mucosal 60 Acral 0 |
Mucosal 60 Acral 0 |
NS | NS |
Abbreviations: ORR, overall response rate; DCR, disease control rate; PFS, progression-free survival; OS, overall survival; NS, not specified. a Included patients with KIT amplifications; b only patients with KIT mutations; c considering 22 patients in part II with KIT mutations; d included three patients with KIT amplifications; e after prior treatment with imatinib; f patients without central nervous system metastases; g patients with central nervous system metastases.