Table 2.
Major characteristics and clinical outcomes in patients receiving a phase I trial therapy (n = 27)
| Age | Pathology | Prior therapy | OS | BMI | MDACC score | Phase I trials | PFS | CMS46 or Foundation Med |
|---|---|---|---|---|---|---|---|---|
| Vaginal cancer | ||||||||
| 45 | A | 2 | 41.9 | 33.2 | 1 | Bevacizumab and Temsirolimus | 1.0 | ND |
| 81 | S | 0 | 5.8 | 21.9 | 0 | Gemcitabine and Dasatinib | 0.9 | ND |
| 61 | M | 4 | 2.0 | 23.0 | 3 | PI3K Inhibitor and Paclitaxel | 0.9 | ND |
| 59 | A | 3 | 4.8 | 37.8 | 2 | Bevacizumab and Temsirolimus plus Carboplatin | PFS1 = 1.4 | ND |
| CHK1 Inhibitor | PFS2 = 1.5 | |||||||
| Erlotinib and Pralatrexate | PFS3 = 2.2 | |||||||
| 53 | S | 2 | 12.9 | 18.6 | 0 | Aurora Kinase Inhibitor | 4.9 | ND |
| 57 | S | 1 | 1.8 | 22.9 | 2 | Trientine and Carboplatin | 0.7 | PIK3CA (E545K) |
| 57 | S | 0 | 28+ | 21.3 | 1 | Everolimus and Pazopanib | PFS1 = 18.2 | PIK3CA (E545K), PTPRD (S1845fs*2) and STK11 loss |
| PI3K Inhibitor | PFS2 = 1.9 | |||||||
| 72 | M | 1 | 4.3 | 21.0 | 1 | Ipilimumab and Imatinib | 2.3 | PTEN loss, C17orf39, KDR, KIT and MYST3 amplification |
| 58 | S | 1 | 15+ | 19.5 | 1 | Erlotinib and Pralatrexate | 14.6+ | ERBB2 (S310F), ERBB4 (D609N), FBXW7 (R479Q), RB1 (E539*), ARID2 (Q1194*) and amplification of EPHBI, PIK3CA and SOX2 |
| 67 | A | 2 | 8.4 | 31.3 | 1 | Anastrozole and Everolimus | 2.6 | PTEN (210-1G > A), KRAS (G12V), CTNNB1 (D32N), MPL (P106L), and amplification of MCL1, MYC and NFKB1A |
| 52 | S | 1 | 7.1 | 24.0 | 0 | Erlotinib and Valproic Acid | 2.8 | ND |
| Vulvar cancer | ||||||||
| 37 | S | 1 | 3.7 | 21.2 | 2 | PI3K inhibitor plus Caboplatin and Paclitaxel | PFS1 = 1.4 | PIK3CA: mutation not detected |
| Erlotinib and Valporic acid | PFS2 = 0.6 | |||||||
| 58 | S | 1 | 13.2 | 30.5 | 1 | Erlotinib and Valporic acid | PFS1 = 3.9 | BRAF, KRAS and PIK3CA: no mutation detected |
| Bevacizumab and Cetuximab plus Erlotinib | PFS2 = 7.2 | |||||||
| 74 | S | 1 | 6.4 | 23.7 | 1 | Microtube Inhibitor | 3.1 | ND |
| 60 | M | 6 | 4.4 | 22.3 | 2 | PI3K Inhibitor | 2.0 | Single Gene: c-KIT (L576P) |
| 42 | S | 1 | 2.0 | 23.7 | 1 | Bevacizumab and Trastuzumab plus Lapatinib | 0.7 | ND |
| 42 | S | 0 | 1.5 | 19.9 | 2 | Src Inhibitor | 0.5 | ND |
| 78 | S | 0 | 20.3 | 35.5 | 1 | Erlotinib and Valporic Acid | 6.1 | ND |
| 37 | S | 1 | 1.7 | 18.7 | 2 | Camptothecin | 0.8 | ND |
| 55 | S | 3 | 4.8 | 15.8 | 1 | Histone Deacetylase Inhibitor | 0.5 | ND |
| 41 | S | 0 | 2.2 | 18.8 | 2 | Sirolimus and Docetaxel | 1.7 | ND |
| 54 | S | 1 | 2.9 | 26.6 | 1 | Lapatinib and Sirolimus | 1.5 | Single Gene: BRAF (V600E) |
| 60 | M | 2 | 4.6 | 22.9 | 3 | Multikinase Inhibitor | 2.9 | ND |
| 69 | S | 1 | 22.6 | 30.9 | 2 | Carboplatin and Trientine | 0.9 | A 46-gene panel: no mutation detected |
| 33 | S | 1 | 10.0 | 24.8 | 2 | Lenalidomide and Temsirolimus | 1.9 | ND |
| 73 | S | 1 | 5.6 | 22.4 | 2 | Crizotinib and Pazopanib | 1.6 | KRAS (R102T), TET2 (W1198*), TP53 (R248Q), and CDK2NA/B loss |
| 55 | M | 1 | 3.4 | 24.2 | 2 | Translation Initiation Inhibition | 1.0 | ND |
OS overall survival, BMI body mass index, MDACC score the sum of five variables (low serum albumin, high serum lactate dehydrogenase, ECOG performance status of 1 or higher more than two metastatic sites, and gastrointestinal tumor type), PFS progression-free survival (1, 2, or 3 indicates the first, second, or third line of phase I trial), * deletion , A adenocarcinoma, S squamous cell carcinoma, M melanoma, ND not done