Treatment options based on dominant AML mutations
| Dominant mutation . | Available target agent . | Available therapeutic regimens . | Therapeutic impact . |
|---|---|---|---|
| FLT3-ITD or TKD | Midostaurin | In combination with “7 + 3” | Incremental improvement in survival |
| Gilteritinib | Single-agent therapy in relapse | Single-agent response rate of 34%8 | |
| In combination with higher doses of cytarabine | Higher response rate in combination9 | ||
| In combination with hypomethylating agents | |||
| Quizartinib | Single-agent maintenance; single-agent therapy in relapse; combination therapy at diagnosis | ||
| IDH1 | Ivosidenib | Single agent OR in combination with “7 + 3” OR in combination with hypomethylating agents at diagnosis | Response rate with hypomethylating agents in newly diagnosed AML was 47%, and 1-y event-free survival was 37%10 |
| IDH2 | Enasidenib | Single agent in relapse | Median overall survival in relapsed setting was 6.5 mo11 |
| Dominant mutation . | Available target agent . | Available therapeutic regimens . | Therapeutic impact . |
|---|---|---|---|
| FLT3-ITD or TKD | Midostaurin | In combination with “7 + 3” | Incremental improvement in survival |
| Gilteritinib | Single-agent therapy in relapse | Single-agent response rate of 34%8 | |
| In combination with higher doses of cytarabine | Higher response rate in combination9 | ||
| In combination with hypomethylating agents | |||
| Quizartinib | Single-agent maintenance; single-agent therapy in relapse; combination therapy at diagnosis | ||
| IDH1 | Ivosidenib | Single agent OR in combination with “7 + 3” OR in combination with hypomethylating agents at diagnosis | Response rate with hypomethylating agents in newly diagnosed AML was 47%, and 1-y event-free survival was 37%10 |
| IDH2 | Enasidenib | Single agent in relapse | Median overall survival in relapsed setting was 6.5 mo11 |