Table 3.
Treatment outcome based on leukemia cell subtype and minimal residual disease in bone marrow at Day 42 (end of induction)
| Number of patients | 5-year EFS, % (95% CI) | 5-year CRR, % (95% CI) | |||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| MRD <0.01 |
MRD 0.01% to<1% | MRD ≥1% |
MRD <0.01% |
MRD 0.01% to<1% | MRD ≥1% |
P-value | MRD <0.01% |
MRD 0.01% to<1% |
MRD ≥1% |
P-value | |
| ETV6-RUNX1 | 115 (91.3) | 11 (8.73) | 0 (0.00) | 99.1 (97.1–100) |
100 (100–100) |
---- | 0.76 | 0 | 0 | ---- | ---- |
| High-hyperdiploid | 145 (94.8) | 8 (5.23) | 0 (0.00) | 96.0 (92.1–99.9) |
80.0 (48.6–100) |
---- | 0.20 | 2.5 (0.0–5.4) |
20.0 (0.0–59.2) |
---- | 0.07 |
| DUX4-rearranged | 19 (95.0) | 1 (5.00) | 0 (0.00) | 94.7 (83.3–100) |
100 (100–100) |
---- | 0.82 | 0 | 0 | ---- | ---- |
| TCF3-PBX1* | 14 (87.5) | 1 (6.25) | 1 (6.25) | 100 (100–100)* | 0 | 0 | 0.006 | 0* | ----* | ----* | 1.00 |
| PAX5alt** | 16 (66.7) | 8 (33.3) | 0 (0.00) | 73.4 (48.7–98.1) |
100 (100–100) |
---- | 0.12 | 26.6 (3.1–50.0) |
0 | ---- | 0.11 |
| T Cell | 76 (82.6) | 9 (9.78) | 7 (7.61) | 87.8 (79.6–96.0) |
66.7 (33.0–100) |
57.1 (20.4–93.8) |
0.05 | 6.6 (1.0–12.2) |
22.2 (0.0–51.3) |
42.9 (2.6–83.1) |
0.01 |
| ETP | 6 (66.7) | 2 (22.2) | 1 (11.1) | 100 (100–100) |
100 (100–100) |
0** | 0.02 | 0 | 0 | 100 | 0.02 |
| iAMP21 | 5 (100) | 0 (0.00) | 0 (0.00) | 80.0 (39.4–100) |
---- | ---- | ---- | 20.0 (0.0–59.2) |
---- | ---- | ---- |
| Hypodiploid | 5 (83.3) | 1 (16.7) | 0 (0.00) | 100 (100–100) |
100 (100–100) |
---- | 0.48 | 0 | 0 | ---- | ---- |
| BCR-ABL1 | 11 (84.6) | 1 (7.69) | 1 (7.69) | 80.8 (56.3–100) |
100 (100–100) |
0 | 0.14 | 10.1 (0.0–30.0) |
0 | 100 (100–100) |
0.09 |
| BCR-ABL1-like† | 9 (60.0) | 3 (20.0) | 3 (20.0) | 77.8 (50.6–100) |
100 (100–100) |
33.3 (0.0–86.6) |
0.13 | 11.1 (0.0–33.1) |
0 | 0 | 0.76 |
| ETV6-RUNX1-like§ | 7 (77.8) | 2 (22.2) | 0 (0.00) | 71.4 (37.9–100) |
50.0 (1.0–99.0) |
---- | 0.48 | 14.3 (0.0–42.7) |
50.0 (0.0–100) |
---- | 0.28 |
| KMT2A-rearranged | 20 (80.0) | 5 (20.0) | 0 (0.00) | 75.0 (53.8–96.2) |
40.0 (0.0–82.9) |
---- | 0.11 | 15.0 (0.0–31.1) |
40.0 (0.0–91.0) |
---- | 0.15 |
| MEF2D-rearranged€ | 3 (100) | 0 (0.00) | 0 (0.00) | 66.7 (23.2–100) | ---- | ---- | ---- | 33.3 (0–98.7) |
---- | ---- | ---- |
| ZNF384-rearranged | 7 (100) | 0 (0.00) | 0 (0.00) | 100 (100–100) |
---- | ---- | ---- | 0 | ---- | ---- | ---- |
| NUTM1-rearranged | 3 (100) | 0 (0.00) | 0 (0.00) | 100 (100–100) |
---- | ---- | ---- | 0 | ---- | ---- | ---- |
| PAX5 P80R | 2 (100) | 0 (0.00) | 0 (0.00) | 100 (100–100) |
---- | ---- | ---- | 0 | ---- | ---- | ---- |
| B-other | 49 (83.1) | 8 (13.6) | 2 (3.39) | 93.6 (86.2–100) |
62.5 (31.9–93.1) |
50 (1.0–99.0) |
0.002 | 2.2 (0.0–6.6) |
37.5 (1.1–73.9) |
50 (0.0–100) |
<.001 |
| Total | 512 (87.2) | 60 (10.2) | 15 (2.6) | 92.6 (89.9–95.3) |
78.9 (66.7–91.1) |
40.0 (12.8–67.2) |
<0.001 | 4.4 (2.5–6.2) |
16.1 (6.2–25.9) |
40.0 (13.9–66.1) |
<.001 |
Abbreviation: MRD, minimal residual disease; No, number of patients; EFS, event-free survival; CCR, cumulative risk of any relapse; CI, confidence interval; ETP, early T-cell precursor ALL.
Among patients TCF3-PBX1 ALL, one with day-42 MRD<0.01% relapsed at 5.7 years, and two with positive MRD died of transplant-related toxicities at 0.6 and 2.4 years, respectively.
Of the 16 PAX5alt patients with day-42 MRD<0.01%, 4 relapsed (two hematological and two CNS relapses).
Of the 9 BCR-ABL1-like patients with day-42 MRD<0.01%, one developed CNS relapse.
Of the 7 ETV6-RUNX1-like patients with day 42-MRD<0.01%, one had hematological relapse, and another developed myelodysplastic syndrome.
Of the three patients with MEF2D-rearranged ALL and day-42 MRD<0.01%, one 12-year old with standard-risk disease died and relapsed at 2.9 years, and the other two patients were alive in remission at 3.6 and 4.0 years, respectively; data shown were 3-year results.