Table 1.
Trials of single agent tipifarnib in acute myelogenous leukemia
| Phase | Patient population | Tipifarniba dose-schedule | Response rate | Author |
|---|---|---|---|---|
| I | Rel/Refa | 100–1200, 21/28 days | CR 8% (2/34) | Karp (2001) |
| MTD 900 | OR 29% (10/34) | |||
| I | Rel/Ref | 400–1400, alternate week | CR 33% (3/9) | Kirschbaum (2007) |
| MTD 1200 | @ MTD | |||
| II | Rel/Ref | 600, 21/28 days | CR 7% (11/169) | Harousseau (2007a) |
| sCR 4% (6/169) | ||||
| med OS 12.2 mo | ||||
| II | New Dx | 600, 21/28–63 days | CR 14% (22/158) | Lancet (2007) |
| Poor risk | OR 23% (37/158) | |||
| Age ≥ 65 | med DFS 7.3 mo | |||
| CR med OS 18.6 mo | ||||
| II | New Dx | 300–600, 21/28 days | CR 6–15% | Erba (2007) |
| Age ≥ 70 | vs alternate week | CR 15%/OR 20% | ||
| @ 300 × 21/28 days | ||||
| III | New Dx | 600 mg, 21/28 days | CR 8% (18/228) | Harousseau (2007b) |
| Poor risk | vs BSC including HU | med DFS 8 mo | ||
| Age ≥ 70 | (vs 0% BSC/HU arm) | CR med OS 20 mob |
amg bid.
bno significant difference between tipifarnib and BSC arms with regard to OS.
Abbreviations: Rel/Ref, relapsed/refractory; CR, complete remission; sCR, sustained complete remission; OR, overall response; MTD, maximal tolerated dose; DFS, disease-free survival; OS, overall survival; BSC, best supportive care; HU, hydroxyurea; mo, months.