Patient characteristics
| Parameter . | Patient 1 . | Patient 2 . | Patient 3 . | Patient 4 . | Patient 5 . | Patient 6 . | Patient 7 . | Patient 8 . | Patient 9 . | Patient 10 . | Patient 11 . | Patient 12 . | Patient 13 . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age at diagnosis (years) | 0.4 | 0.3 | 0.6 | 5.2 | 1.0 | 0.5 | 0.4 | 0.4 | 17.5 | 1.7 | 0.4 | 0.2 | 0.1 |
| Sex | F | F | F | F | M | F | F | F | F | M | M | F | M |
| WBC (initial)/µL | 103.000 | 334.000 | 416.500 | 284.430 | 42.400 | 210.800 | 166.450 | 73.960 | 27.010 | 24.630 | 1023.000 | 733.500 | 221.000 |
| Blasts (PB) initial (%) | 91 | 97 | 97 | 92 | 82 | 88 | 80 | 88 | 80 | 30 | 96 | 94 | 90 |
| Blasts (BM) initial (%) | 97 | 98 | 99 | 95 | 96 | 88 | 95 | 93 | 89 | 72 | 93 | 88 | 95 |
| Central nervous system status | 1 | n.d. | 3 | 2c | n.d. | n.d. | n.d. | 3 | 1 | 2a | 2a | n.d. | n.d. |
| Immunophenotype | Pro-B | Pro-B | Pro-B | Pro-B | c-ALL | Pro-B | c-ALL | c-ALL | Pro-B | Pro-B | Pro-B | Pro-B | Pro-B |
| MLL-fusion partner | ENL | ENL | ENL | AF4 | AF10 | AF9 | AF9 | AF9 | AF4 | AF4 | ENL | AF4 | AF4 |
| Pred-response | Good | Poor | Good | Poor | Good | Good | Poor | Poor | Good | Good | Poor | Poor | Good |
| Blasts (BM), day 15 (%) | 1 | 32 | 3 | 15 | 0.5 | 8 | 1 | 0 | 0 | 1 | 75 | 35 | 6 |
| MRD treatment, day 33 | Neg | Neg | n.d. | n.d. | Neg | n.d. | Neg | n.d. | 10−6 | 10−3 | n.d. | n.d. | 10−6 |
| MRD, second time point* | 10−6† | Neg | n.d. | 10−3 | 10−5 | n.d. | Neg | n.d. | Neg | 10−4 | n.d. | n.d. | 10−6 |
| Relapse | No | Yes | No | Yes | Yes | No | Yes | No | No | No | Yes | Yes | Yes |
| Death | No | Yes | No | Yes | Yes | Yes | Yes | Yes | No | No | Yes | Yes | Yes |
| Study | BFM 2000 | BFM 2000 | IFAN 2006 | BFM 2009 | BFM 2000 | IFAN 2006 | IFAN 2006 | IFAN 2006 | BFM 2009 | BFM 2009 | IFAN 1999 | IFAN 1999 | IFAN 2006 |
| Parameter . | Patient 1 . | Patient 2 . | Patient 3 . | Patient 4 . | Patient 5 . | Patient 6 . | Patient 7 . | Patient 8 . | Patient 9 . | Patient 10 . | Patient 11 . | Patient 12 . | Patient 13 . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age at diagnosis (years) | 0.4 | 0.3 | 0.6 | 5.2 | 1.0 | 0.5 | 0.4 | 0.4 | 17.5 | 1.7 | 0.4 | 0.2 | 0.1 |
| Sex | F | F | F | F | M | F | F | F | F | M | M | F | M |
| WBC (initial)/µL | 103.000 | 334.000 | 416.500 | 284.430 | 42.400 | 210.800 | 166.450 | 73.960 | 27.010 | 24.630 | 1023.000 | 733.500 | 221.000 |
| Blasts (PB) initial (%) | 91 | 97 | 97 | 92 | 82 | 88 | 80 | 88 | 80 | 30 | 96 | 94 | 90 |
| Blasts (BM) initial (%) | 97 | 98 | 99 | 95 | 96 | 88 | 95 | 93 | 89 | 72 | 93 | 88 | 95 |
| Central nervous system status | 1 | n.d. | 3 | 2c | n.d. | n.d. | n.d. | 3 | 1 | 2a | 2a | n.d. | n.d. |
| Immunophenotype | Pro-B | Pro-B | Pro-B | Pro-B | c-ALL | Pro-B | c-ALL | c-ALL | Pro-B | Pro-B | Pro-B | Pro-B | Pro-B |
| MLL-fusion partner | ENL | ENL | ENL | AF4 | AF10 | AF9 | AF9 | AF9 | AF4 | AF4 | ENL | AF4 | AF4 |
| Pred-response | Good | Poor | Good | Poor | Good | Good | Poor | Poor | Good | Good | Poor | Poor | Good |
| Blasts (BM), day 15 (%) | 1 | 32 | 3 | 15 | 0.5 | 8 | 1 | 0 | 0 | 1 | 75 | 35 | 6 |
| MRD treatment, day 33 | Neg | Neg | n.d. | n.d. | Neg | n.d. | Neg | n.d. | 10−6 | 10−3 | n.d. | n.d. | 10−6 |
| MRD, second time point* | 10−6† | Neg | n.d. | 10−3 | 10−5 | n.d. | Neg | n.d. | Neg | 10−4 | n.d. | n.d. | 10−6 |
| Relapse | No | Yes | No | Yes | Yes | No | Yes | No | No | No | Yes | Yes | Yes |
| Death | No | Yes | No | Yes | Yes | Yes | Yes | Yes | No | No | Yes | Yes | Yes |
| Study | BFM 2000 | BFM 2000 | IFAN 2006 | BFM 2009 | BFM 2000 | IFAN 2006 | IFAN 2006 | IFAN 2006 | BFM 2009 | BFM 2009 | IFAN 1999 | IFAN 1999 | IFAN 2006 |
Central nervous system status is as described previously.33
BM, bone marrow; c-ALL, common acute lymphoblastic leukemia; F, female; IFAN, Interfant trial; M, male; n.d., not determined; Neg, negative; PB, peripheral blood; Pro-B, pro-B cell.
Day 78 for BFM patients; before MARMA for IFAN patients.
Low positive, not quantifiable.39