Table 1.
Treatment groups and clinical outcome according to leukemia subtypes
| Subtype | N | Low risk (N=260) N (%) | Standard risk (N=280) N (%) | High risk (N=58) N (%) | Transplant N. | 5-year EFS, % (95% CI) | 5-year OS, % (95% CI) | 5-year CRR, % (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* | 17 | 1 (5.9) | 14 (82.4) | 2 (11.8) | 2 | 88.2(71.7–100) * | 88.2(71.7–100) * | 0* |
| PAX5alt** | 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) |
| iAMP21† | 5 | 1 (20.0) † | 4 (80.0) | 0 (0.00) | 0 | 80.0(39.4–100) † | 100(100–100) † | 20.0(0.0–59.2) |
| Hypodiploid £ | 6 | 1 (16.7) | 4 (66.7) | 1 (16.7) | 1 | 100(100–100) £ | 100(100–100) £ | 0£ |
| 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-likeξ | 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-like§ | 9§ | 2 (22.2) | 7 (77.8) | 0 (0.00) | 0 | 66.7(35.9–97.5) § | 87.5(66.1–100) § | 22.2(0.0–51.3) § |
| 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-r ¥ | 3 | 1 (33.3) | 2 (66.7) | 0 (0.00) | 0 | 66.7 (23.2–100) ¥ | 66.7(23.2–100) ¥ | 33.3(0.0–98.7) ¥ |
| ZNF384-r € | 7 | 0 (0.00) | 7 (100) | 0 (0.00) | 0 | 100(100–100) | 100(100–100) | 0 |
| NUTM1-r € | 3 | 0 (0.00) | 3 (100) | 0 (0.00) | 0 | 100(100–100) | 100(100–100) | 0 |
| PAX5 P80R € | 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) |
Abbreviation: No, number of patients; EFS, event-free survival; OS, overall survival; CCR, cumulative risk of any relapse; CI, confidence interval; ETP, early T-cell precursor ALL.
one standard-risk with day-42 MRD<0.01% relapsed at 5.7 years and 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.
four patients with day-42 MRD<0.01% relapsed.
one low-risk patient with day 42 MRD<0.01% relapsed at 3.4 years and remained in second remission for 5.6 years.
one 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).
two patients had treatment-related death and one died of multiple secondary malignancies.
two standard-risk patients relapseds and one low-risk patient developed secondary myelodysplastic syndrome.
two patients were alive in remission at 3.6 and 4.0 years, respectively, and one 12-years old standard-risk patient with day 42 MRD<0.01% died of relapse at 2.9 years; data shown was 3-year result.
remission durations for the 7 patients with ZNF384-rearranged ALL were 6.8, 7.8, 9.4, 9.7, 10.3, 11.1, and 11.5 years; for the 3 with NUTM1-rearranged ALL 4.4, 4.7, 7.0 years, and for the 2 with PAX5 P80R 7.1 and 9.1 years, respectively.