Table 1.
Clinical trials for targeted therapies in patients with mIDH AML a.
| Phase b | Treatment Setting c | Intervention | No. of Patients d | Age, y | ORR, % e | Combined CR/CRi/CRp, % | Time to CR, mo | OS, mo (Median, 95% CI) f | Reference |
|---|---|---|---|---|---|---|---|---|---|
| Enasidenib | |||||||||
| 1/2 | R/R | Enasidenib monotherapy | 109 | 67 (19–100) | 38.5 | 26.6 | 3.7 (0.7–11.2) | 9.3 | [91,92] |
| Front-line | 37 | 77 (58–87) | 37.8 | 18.9 | 5.6 (3.4–12.9) | 10.4 (5.7–15.1) | [92,93] | ||
| 1 | Front-line | Enasidenib plus 7 + 3 g | 38 | 63 (32–76) | 81 | 62 | NR h | NR | [94,95] |
| 1/2 | Front-line | Enasidenib plus 5-Aza | 6 | 68 (64–79) | 50 | 33.3 | NR | NR | [96,97] |
| 3 | R/R | Enasidenib monotherapy vs BSC i, 5-Aza, or cytarabine | No results available | [98] | |||||
| Ivosidenib | |||||||||
| 1 | R/R | Ivosidenib monotherapy | 125 | NR | 41.6 | 30.4 | NR | NR | [99,100] |
| 1 | Front-line | Ivosidenib plus 7 + 3 | 27 | 60 (24–76) | 83 | 70 | NR | NR | [94,95] |
| 1/2 | Front-line | Ivosidenib plus 5-Aza | 5 | 81 (72–88) | 60 | 60 | NR | NR | [96,97] |
| 3 | Front-line | Ivosidenib plus 5-Aza vs. 5-Aza plus placebo | No results available | [101] | |||||
| Venetoclax | |||||||||
| 2 j | R/R | Venetoclax monotherapy | 12 | 71 (19–84) k | NR | 33 | NR | NR | [102] |
| 1 | Front-line | Venetoclax plus 5-Aza or DAC | 17 | ≥65 l | 77 | 59 | NR | NR | [103,104] |
a Median (range), unless noted otherwise; b Trials ongoing unless noted otherwise; c R/R: Relapsed/refractory; d Response-evaluable with mIDH; e CR, CRi, complete response with incomplete platelet recovery (CRp), partial remission, or morphologic leukemia-free state; f OS: Overall survival; CI: Confidence interval; g 7 + 3: anthracycline for three days and continuous infusion of cytarabine for seven days; h BSC: Best supportive care; i NR: Not reported; j Completed; k Includes non-mIDH patients; l Median age not reported.