Table 1.
Patient characteristics
| Patients (n = 31) | n (% or range) |
|---|---|
| Sex | |
| Male | 18 (58.1) |
| Female | 13 (41.9) |
| Diagnosis | |
| Advanced MDS | 4 (23.5) |
| Relapsed AML | 11 (35.5) |
| Refractory AML | 7 (22.6) |
| Postcytotoxic therapy MDS/AML | 9 (29.0) |
| Age, y, median (range) | 10.2 (1.3-17.4) |
| Extramedullary disease | |
| No | 29 (93.5) |
| Yes | 2 (6.5) |
| BM blast at diagnosis, median (range) | 25% (0-95) |
| BM blasts before the first venetoclax therapy, median (range) | 20% (0-80) |
| Previous lines of therapies since last complete response, median (range) | 3 (1-7) |
| 0 | 4 (23.5) |
| 1 | 3 (17.6) |
| 2 | 2 (11.8) |
| ≥ 2 | 8 (45.1) |
| Previous HSCT | |
| 0 | 18 (58.1) |
| 1 | 9 (29.0) |
| 2 | 4 (12.9) |
| Unfavorable genetics | |
| KMT2A rearrangements | 8 (25.8) |
| - 7/del7q | 6 (19.3) |
| FLT3-ITD | 5 (16.1) |
| Complex karyotype | 2 (6.5) |
| KRAS | 2 (6.5) |
| PTPN11 | 1 (3.2) |
| No. of venetoclax cycles per patient, median (range) | 2 (1-15) |
| Best response achieved | |
| CR∗ | 16 (51.6) |
| PR | 6 (19.4) |
| ORR | 22 (71.0) |
| NR | 8 (25.8) |
| TF | 1 (3.2) |
| No. of cycles to best response (in 22 responders), median, range | 1 (1-7) |
ORR, overall response rate.
∗
Five with morphological CR; 4 who tested flow cytometry-minimal residual disease negative; 2 who tested polymerase chain reaction-minimal residual disease negative (with extramedullary complete remission detected upon fluorodeoxyglucose-positron emission tomography and skin biopsy in 1 case); and 5 with CR with incomplete recovery.