Table 1.
Treatment groups and clinical outcome according to leukemia subtypes
| Low risk (N = 260) | Standard risk (N = 280) | High risk(N = 58) | Transplant N | 5-year EFS, % | 5-year OS, % | 5-year CRR, % | ||
|---|---|---|---|---|---|---|---|---|
| Subtype | N | N (%) | N (%) | N (%) | (95% CI) | (95% CI) | (95% CI) | |
| ETV6–RUNX1 | 128 | 111 (86.7) | 17 (13.3) | 0 (0.00) | 0 | 98.4 (95.9–100) | 99.2 (97.4–100) | 0.8 (0.0–2.3) |
| Hyperdiploid | 154 | 103 (66.9) | 51 (33.1) | 0 (0.00) | 0 | 95.3 (91.2–99.4) | 99.4 (97.8–100) | 3.3 (0.1–6.5) |
| DUX4-rearranged | 20 | 8 (40.0) | 12 (60.0) | 0 (0.00) | 0 | 95.0 (84.2–100) | 95.0 (84.2–100) | 0 |
| TCF3–PBX1 a | 17 | 1 (5.9) | 14 (82.4) | 2 (11.8) | 2 | 88.2 (71.7–100)a | 88.2 (71.7–100)a | 0a |
| PAX5altb | 24 | 4 (16.7) | 20 (83.3) | 0 (0.00) | 0 | 82.7 (65.3–100) | 100 (100–100) | 17.3 (1.5–33.1) |
| T-cell | 94 | 0 (0.00) | 79 (84.0) | 15 (16.0) | 11 | 81.3 (72.5–90.1) | 88.2 (80.8–95.6) | 12.0 (5.3–18.7) |
| ETP | 10 | 0 (0.00) | 0 (0.00) | 10 (100) | 6 | 80.0 (53.5–100) | 77.1 (49.9–100) | 20.0 (0.0–46.1) |
| iAMP21c | 5 | 1 (20.0)c | 4 (80.0) | 0 (0.00) | 0 | 80.0 (39.4–100)c | 100 (100–100)c | 20.0 (0.0–59.2) |
| Hypodiploidd | 6 | 1 (16.7) | 4 (66.7) | 1 (16.7) | 1 | 100 (100–100)d | 100 (100–100)d | 0d |
| BCR–ABL1 | 13 | 0 (0.00) | 0 (0.00) | 13 (100) | 0 | 76.2 (51.9–100) | 83.1 (60.8–100) | 16.2 (0.0–37.7) |
| BCR–ABL1-likee | 15 | 3 (20.0) | 9 (60.0) | 3 (20.0) | 2 | 73.3 (47.0–99.6) | 86.7 (66.1–100) | 6.7 (0.0–19.9) |
| ETV6–RUNX1-likef | 9f | 2 (22.2) | 7 (77.8) | 0 (0.00) | 0 | 66.7 (35.9–97.5)f | 87.5 (66.1–100)f | 22.2 (0.0–51.3)f |
| KMT2A-r | 28 | 0 (0.00) | 18 (64.3) | 10 (35.7) | 1 | 64.1 (43.9–84.3) | 75.0 (56.0–94.0) | 25.2 (8.7–41.7) |
| MEF2D-rg | 3 | 1 (33.3) | 2 (66.7) | 0 (0.00) | 0 | 66.7 (23.2–100)g | 66.7 (23.2–100)g | 33.3 (0.0–98.7)g |
| ZNF384-rh | 7 | 0 (0.00) | 7 (100) | 0 (0.00) | 0 | 100 (100–100) | 100 (100–100) | 0 |
| NUTM1-rh | 3 | 0 (0.00) | 3 (100) | 0 (0.00) | 0 | 100 (100–100) | 100 (100–100) | 0 |
| PAX5 P80Rh | 2 | 2 (100) | 0 (0.00) | 0 (0.00) | 0 | 100 (100–100) | 100 (100–100) | 0 |
| B other | 60 | 23 (38.3) | 33 (55.0) | 4 (6.67) | 2 | 86.3 (76.9–95.7) | 93.3 (86.4–100) | 10.3 (2.4–18.2) |
| Total | 598 | 260 (43.5) | 280 (46.8) | 58 (9.70) | 25 | 88.8 (85.9–91.7) | 94.0 (91.8–96.2) | 7.4 (5.3–9.6) |
Abbreviations: CI, confidence interval; CRR, cumulative risk of any relapse; EFS, event-free survival; ETP, early T-cell precursor ALL; iAMP21, intrachromosomal amplification of chromosome 21; OS, overall survival.
aOne standard-risk patient with day 42 MRD <0.01% relapsed at 5.7 years and was alive in second remission for 2.1 years, and two high-risk patients died of transplant-related toxicities at 0.6 and 2.4 years, respectively.
bFour patients with day 42 MRD <0.01% relapsed.
cOne low-risk patient with day 42 MRD <0.01% relapsed at 3.4 years and remained in second remission for 5.6 years.
dOne patient with day 42 MRD <0.01% developed secondary acute myeloid leukemia at 5.8 years, resulting in 7-year EFS of 75.0% (23.1–100).
eTwo patients had treatment-related death, and one died of multiple secondary malignancies.
fTwo standard-risk patients relapsed, and one low-risk patient developed secondary myelodysplastic syndrome.
gTwo patients were alive in remission at 3.6 and 4.0 years, respectively, and one 12-year-old standard-risk patient with day 42 MRD <0.01% died of relapse at 2.9 years; data shown are 3-year results.
hRemission durations for the seven patients with ZNF384-rearranged ALL were 6.8, 7.8, 9.4, 9.7, 10.3, 11.1, and 11.5 years; for the three with NUTM1-rearranged ALL, 4.4, 4.7, and 7.0 years; and for the two with PAX5 P80R, 7.1 and 9.1 years, respectively.