Table 1. Phase I and phase II studies of VPA.
| Phase I/II studies | ||||
| Author | Malignancy; N | Combination | Dosage | Response rate (RR) |
| Garcia-Manero et al [53] | AML/ myelodysplastic syndrome (MDS) (54) | 5AZA | 50 mg/kg/day per os (PO) | RR 22% |
| Soriano et al, [44] | AML/MDS (53) | All-Trans Retinoic AcidIV: intravenous (ATRA) + 5AZA | 50 mg/kg/day PO 7 days | RR 47% |
| Daud et al [54] | Melanoma (39) | Karenitecine | 75 mg/kg/day IV d1 to d5 | 47% stable disease |
| Rocca et al [45] | Melanoma (29) | Dacarbazine + interferon | Serum level of 50 to 125 mg/dL | 5% CR; 11% PR; 16% stable disease |
| Munster et al [25] | Solid tumours(44) | Epirrubicine/ 5-fluorouracil, epirrubicin, cyclophosphamide (FEC) | 120 mg/kg IV | RR 22% (phase I) RR 68% phase II (breast cancer) |
| Iwahashi et al [17] | Pancreas/biliary(12) | S-1 | 15 mg kg 2xd PO | 8% PR; 83% stable disease |
| Phase II studies | ||||
| Author | Malignancy; N | Combination | Dosage | Response rate |
| Kuendgen et al [55] | AML/MDS (75) | ATRA | Serum levels 50 to 100 mcg/mL | RR 5% (AML), 16% (MDS) |
| Pilatrino et al [56] | AML/MDS (20) | ATRA | Serum levels 45 to 100 mcg/mL | Clinical benefit 30% |
| Candelaria et al [57] | Solid tumours (17) | Hidralazine + chemotherapy | 40 mg/kg/day | 0% CR, 27% PR, 53% stable disease |
| Raffoux et al [58] | AML/MDS (65) | 5-AZA + ATRA | 35–50 mg/kg for 7 days | RR 26% |
| Mohammed et al [22] | Neuroendocrine tumours (8) | - | 500 mg 2xd PO; non significant (NS) 50–100 mcg/mL | 12% PR, 50% stable disease |
| Krauze et al [16] | Glioblastoma multiforme (37) | Temozolomide + radiotherapy | 25 mg/kg/day | 81% stable disease |
| Issa et al [15] | AML/MDS (149) | ± Decitabine | 50 mg/kg 7 days | RR 51% versus 58% combination |