Table 2.
Clinical trials containing single agent treatment with FLT3 inhibitors (FLT3i).
| FLT3 Inhibitor | AML Setting | Patients (n) | FLT3 Mutation | Phase | Response, n CRc (%) PR (%) |
LFS (mo) | OS (mo) | Ref. |
|---|---|---|---|---|---|---|---|---|
| Midostaurin | r/r AML AML 1st line |
17 2 |
ITD 18 TKD 1 |
2 | 0 (0) 1 (5.2) |
n.a. | n.a. | [52] |
| r/r AML | 35 | ITD 26 TKD 9 |
1 | 0 (0) 1 (2.9) |
n.a. | 3.3 | [53] | |
| Sorafenib | r/r AML | 13 | ITD 12 ITD + TKD 1 |
2 | 6 (46.2) n.a. |
2.4 | n.a. | [54] |
| r/r AML | 65 | ITD 65 no TKD |
Survey | 25 (38) | no ASCT: 4.5 prior ASCT: 6.5 |
n.a. | [40] | |
| Crenolanib | r/r AML |
34 | ITD and TKD |
2 | 4 (12%) 1 (3%) |
n.a. | 4.4 | [49] |
| r/r AML Cohort A (no prior TKI) |
18 | ITD 9 TKD 6 ITD + TKD 3 |
2 | 7 (39%) 2 (11%) |
n.a. | 7.8 | [56] | |
| Quizartinib | r/r AML | 76 | ITD 76 no TKD |
2 | 36 (47.4) 14 (18.4) |
5.3 | 22.6 | [47] |
| r/r AML | 245 (allocated to Quizartinib) |
ITD 245 no TKD |
3 | 118 (48) 51 (21) |
n.a. | 6.2 | [46] | |
| Gilteritinib | r/r AML | 191 | ITD 162 TKD 16 ITD + TKD 13 |
1–2 | 70 (37) 23 (12) |
n.a. | 30.0 | [57] |
| r/r AML | 247 (allocated to Gilteritinib) |
ITD 215 TKD 21 ITD + TKD 7 Other 4 * |
3 | 134 (54.3) 33 (13.4) |
4.4 | 9.3 | [50] |
Abbreviations: ASCT, allogeneic stem cell transplantation; CRc—composite complete remission; FLT3i, FLT3 inhibitor; ITD, internal tandem duplication; LFS—Leukemia-free survival, OS—Overall survival TKD, tyrosine kinase domain; PR, partial remission; r/r AML, relapsed or refractory AML; * four patients with unconfirmed FLT3 mutation were assigned to the gilteritinib group.