Table 2.
Outcome measures in pediatric AMkL per Children’s Oncology Group trial
| AAML0531 | AAML1031 | |||||||
|---|---|---|---|---|---|---|---|---|
| Variable | N | 5-year EFS | 5-year OS | 5-year RR from end of course 1b | N | 5-year EFS | 5-year OS | 5-year RR from end of course 1b |
| Morphology | ||||||||
| All M7 FAB | 48 | 62 ± 14% ^ | 64 ± 14% # | 31 ± 17% | 59 | 37 ± 13% ^ | 45 ± 14% *,# | 45 ± 16% |
| M0-M6 FAB | 816 | 53 ± 3% | 66 ± 3% | 36 ± 4% | 1012 | 47 ± 3% | 66 ± 3% * | 43 ± 4% |
| FAB M7 Karyotype | ||||||||
| Not complex | 20 | 53 ± 22% | 53 ± 22% | 55 ± 34% | 31 | 39 ± 18% | 38 ± 18% | 38 ± 22% |
| Complex | 24 | 71 ± 18% | 75 ± 17% | 24 ± 20% | 25 | 36 ± 20% | 53 ± 22% | 49 ± 26% |
| FAB M7 fusion-based classification a | ||||||||
| AMkL, NOS | 4 | - | - | - | 7 | 43 ± 37% | 42 ± 37% | 25 ± 50% |
| AMkL with RBM15::MRTFA fusion | 7 | 86 ± 26% | 86 ± 26% | 0 ± 0% | 14 | 50 ± 27% | 54 ± 14% | 38 ± 29% |
| AMkL with CBFA2T3::GLIS2 | 7 | 43 ± 37% | 43 ± 37% | - | 10 | 10 ± 19% | 10 ± 19% | 60 ± 56% |
| AMkL with NUP98 fusion | 3 | 100 ± 0% | 100 ± 0% | 0 ± 0% | 8 | 30 ± 35% | 42 ± 44% | - |
| AMkL with KMT2A fusion | 3 | - | - | 0 ± 0% | 5 | 40 ± 44% | 40 ± 44% | 25 ± 50% |
| AMkL with (other) myelodysplasia-related changes | 20 | 75 ± 20% | 80 ± 19% | 18 ± 19% | 10 | 58 ± 32% | 69 ± 30% | 35 ± 34% |
Abbreviations: AMkL, acute myeloid leukemia with megakaryocytic differentiation; EFS, event-free survival; FAB, French-American-British classification; N, number; NOS, not otherwise specified; OS, overall survival; RR, relapse risk
a
Excluding those with unknown cytogenetics or fusions identified in ≤2 cases (MECOM, FUS::ERG, and TEC::MLLT10)
b
Including only those with a complete remission
*
AAML1031 M7 significantly worse compared to AAML1031 M0-M6 FAB group (p=0.001)
^
AAML0531 M7 5-year EFS significantly better compared to AAML1031 M7 group (p=0.009)
#
AAML0531 M7 5-year OS better compared to AAML1031 M7 group but not significantly (p=0.069)
- 5-year estimate is undefined