Table 5. Clinical outcomes of CUP patients treatment on a phase I therapy.
| Pt # | Age/Sex | Phase I Regimen | Therapy matched to molecular aberration | Best Response per RECIST | Phase I PFS (m) |
|---|---|---|---|---|---|
| 1 | 22/M | Not treated* | - | - | - |
| 2 | 60/F | A novel PI3K inhibitor | Yes | SD | 5.9 |
| 3 | 21/M | Vandetanib + everolimus | No | PD overall (SD of target lesions, but developed a new liver lesion) | 2.0 |
| 4 | 25/F | Not treated* | - | - | - |
| 5 | 36/F | Carboplatin + bevacizumab + temsirolimus | Yes | SD (On study) | 8.0+ |
| 6 | 54/M | Not treated* | - | - | - |
| 7 | 75/F | Everolimus + anastrozole | Yes | PD | 3.3 |
| 8 | 74/M | Sirolimus + hydroxychloroquine | Yes | SD | 4.5 |
| 9 | 50/M | Lenalidomide + temsirolimus | Yes | PD | 1.4 |
| 10 | 51/F | Not treated* | - | - | - |
| 11 | 73/M | Not treated* | - | - | - |
| 12 | 28/F | Crizotinib + pazopanib | No | SD (On study) | 8.9+ |
| 13 | 67/F | Vandetanib + everolimus | No | PD | 2.7 |
| 14 | 57/F | Liposomal doxorubicin + bevacizumab + temsirolimus | Yes | PD overall (SD of lung lesions, PD of liver lesion) | 2.8 |
| 15 | 65/M | Not treated* | - | - | - |
| 16 | 74/M | Everolimus + denosumab | No | PD | 1.5 |
| 17 | 48/M | Lapatinib + sirolimus | Yes | PD (mixed response) | 1.7 |
* Not treated on a phase I trial due to decision to pursue alternate therapies.
Abbreviations: PD progressive disease, PI3K phosphatidylinositol 3-kinase, PFS progression free survival, RECIST Response Evaluation Criteria In Solid Tumors, SD stable disease.