Table 2.
Results of recent ATO-inclusive trials
| Risk category | Protocol (published trial analysis period) | Median follow-up (years) | No | Age restriction (years) | CR | ED | CIR | Death in CR | DFS | EFS | OS | Therapy-related neoplasm |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Low-intermediate risk |
APL0406142 ATRA-ATO (4-yr) |
3.4 | 127 | 18–71 | 100% | 0% | 1.9% | 0.8% | ns | 97.3% | 99.2% | 0 |
|
APL0406 ATRA-Ida (4-yr) |
3.4 | 132 | 18–71 | 97% | 3% | 13.9% | 3.7% | ns | 80% | 92.6% | 2 | |
|
UK AML1740 ATRA-ATO ± GO (4-yr) |
2.5 | 86 | ≥16 | 94% (all risk groups) | 4% | 1%a,c 0%b,c |
2%c | 97%a 98%b |
92% | 95%d | 0 | |
|
UK-AML17 ATRA-Ida (4-yr) |
2.5 | 92 | ≥16 | 89% (all risk groups) | 6% | 18%a 27%b |
1%c | 78%a 70%b |
71% | 90%c | 1-risk group ns | |
|
MD Anderson139 ATRA/ATO ± GO (3-yr) |
1.9 | 56 | ns | 95% | 3.6% | 0% | 4% of all patients | ns | 89%e | 89%e | ns | |
|
North American Intergroup C971036 No ATO in Consolidation (3-yr) |
2.4 | 144 | ≥15 | 93%–94% | 4% | ns | ns | 77%e | 71%e | ns | ns | |
|
North American IntergroupC9710 ATO in consolidation (3-yr) |
2.4 | 189 | ≥15 | 93%–94% | 4% | ns | ns | 90%e | 84%e | ns | ns | |
|
APML441 ATRA-ATO-Ida (5-yr) |
4.2 | 101 | >1 | 96% | 2% | 5% | 0% | 96% | 92% | 96% | ns | |
| High-risk |
UK AML1740 ATRA-ATO (4-yr) |
2.5 | 30 | ≥16 | ns | ns | ns | 2%c | ns | 87%f | 87%d | ns |
|
UK AML17 ATRA-Ida (4-yr) |
2.5 | 27 | ≥16 | ns | ns | ns | 1%c | ns | 64%f | 84%d | ns | |
|
MD Anderson139 ATRA/ATO ± GO (3-yr) |
1.9 | 26 | ns | 81% | 19% | 11% | 4% of all patients | ns | 65%e | 75%e | ns | |
|
North American Intergroup C971036 No ATO (3-yr) |
2.4 | 58 | ≥15 | 71% | 20% | ns | ns | 46%e | 39%e | ns | ns | |
|
North American Intergroup C9710 ATO consolidation (3-yr) |
2.4 | 55 | ≥15 | 71% | 20% | ns | ns | 87%e | 64%e | ns | ns | |
|
APML441 ATRA-ATO-Ida (5-yr) |
4.2 | 23 | >1 | 88% | 8.7% | 5% | 0% | 95% | 83% | 87% | ns |
Notes:
a
Morphological,
b
molecular,
c
difference between high and low-intermediate risk not given,
d
no significant difference in OS between ATRA-ATO and ATRA/Ida therapy within risk groups,
e
estimated from published survival curves (data not given),
f
no significant difference in EFS between ATRA-ATO and ATRA/Ida in high-risk patients. The year given in parentheses is the time-point at which the survival endpoints were analyzed.
Abbreviations: ATO, arsenic trioxide; CR, complete remission; CIR, cumulative incidence of relapse; DFS, disease-free survival; ED, early death; EFS, event-free survival; OS, overall survival; ATRA, all trans-retinoic acid; ns, not specified; Ida, idarubicin; GO, gemtuzumab ozogamicin.