Key findings and publications related to specific genomically defined targeted therapies for AML patients
| Target . | Therapy . | Key finding or NCTN number if trial is in progress . | Reference . |
|---|---|---|---|
| FLT3 | Midostaurin | Midostaurin with induction chemotherapy results in a survival benefit for chemotherapy-eligible patients with an FLT3 mutation compared with patients who only receive chemotherapy | 12 |
| FLT3 | Quizartinib | Quizartinib as a single agent in relapsed and refractory FLT3+ AML results in a 50% remission rate | 17 |
| FLT3 | Gilteritinib | Gilteritinib as a single agent in relapsed and refractory FLT3+ AML results in a 37% remission rate | 19 |
| FLT3 | Crenolanib | Induction chemotherapy with midostaurin vs crenolanib in FLT3+ AML patients; NCT03258931 | N/A |
| IDH2 | Enasidenib | Enasidenib as a single agent in IDH2+ patients results in a 40% overall response rate with a median OS of 9 mo | 23 |
| IDH1 | Ivosidenib | Ivosidenib as a single agent in IDH1+ patients results in a 40% overall response rate with a median OS of 9 mo | 25 |
| KIT | Dasatinib | Chemotherapy with dasatinib for core binding factor AML patients was tolerated and may improve efficacy; a randomized trial is ongoing (NCT02013648) | 34 |
| Splice gene mutations | H3B-8800 | NCT02841540 | N/A |
| Target . | Therapy . | Key finding or NCTN number if trial is in progress . | Reference . |
|---|---|---|---|
| FLT3 | Midostaurin | Midostaurin with induction chemotherapy results in a survival benefit for chemotherapy-eligible patients with an FLT3 mutation compared with patients who only receive chemotherapy | 12 |
| FLT3 | Quizartinib | Quizartinib as a single agent in relapsed and refractory FLT3+ AML results in a 50% remission rate | 17 |
| FLT3 | Gilteritinib | Gilteritinib as a single agent in relapsed and refractory FLT3+ AML results in a 37% remission rate | 19 |
| FLT3 | Crenolanib | Induction chemotherapy with midostaurin vs crenolanib in FLT3+ AML patients; NCT03258931 | N/A |
| IDH2 | Enasidenib | Enasidenib as a single agent in IDH2+ patients results in a 40% overall response rate with a median OS of 9 mo | 23 |
| IDH1 | Ivosidenib | Ivosidenib as a single agent in IDH1+ patients results in a 40% overall response rate with a median OS of 9 mo | 25 |
| KIT | Dasatinib | Chemotherapy with dasatinib for core binding factor AML patients was tolerated and may improve efficacy; a randomized trial is ongoing (NCT02013648) | 34 |
| Splice gene mutations | H3B-8800 | NCT02841540 | N/A |
N/A, not available; NCTN, National Clinical Trials Network.