Clinical and survival data for EBF1-PDGFRB–positive patients
| Patient No. . | Trial . | Age (y) . | Sex . | WBC × 109/L . | CR . | Slow early responder* . | Postinduction MRD level (%) . | Treatment allocation† . | First remission transplant . | Relapse . | Months to relapse . | Relapse therapy . | Status . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | ALL 97 | 6 | F | 212 | Yes | Yes‡ | ND | Standard therapy | BM and CNS | 31 | Allogeneic transplant | Died of relapse (5 y) | |
| 2 | ALL 97/99 | 12 | F | 17 | Yes | Yes | ND | Regimen B→C | No | Alive (7.6 y) | |||
| 3 | ALL 97/99 | 12 | F | 5 | Yes | Yes | ND | Regimen B→C | BM | 39 | ALLR3, intermediate risk, mitoxantrone, MUD | Alive (7.4 y) | |
| 4 | UKALL 2003 | 13 | F | 60 | Yes | Yes | >10 | Regimen B→C | Allogeneic | BM and bone | 27 | NK | Alive (9.3 y) |
| 5 | UKALL 2003 | 4 | F | 49 | Yes | No | >0.1 | Regimen B→C | No | Alive (7.5 y) | |||
| 6 | UKALL 2003 | 11 | M | 70 | No | Yes | >10 | Regimen B→C | MUD | No | Alive (7.3 y) | ||
| 7 | UKALL 2003 | 19 | M | 8 | Yes | Yes | >10 | Regimen B | CNS | 40 | Allogeneic transplant | Alive (6.8 y) | |
| 8 | UKALL 2003 | 8 | M | 12 | Yes | No | >10 | Regimen A | Skin | 59 | ALLR3, standard risk, mitoxantrone | Alive (6 y) | |
| 9 | UKALL 2003 | 18 | F | 3 | Yes | Yes | >10 | Regimen B→C | Allogeneic | No | Alive (5.2 y) | ||
| 10 | UKALL 2003 | 14 | M | 26 | Yes | Yes | >10 | Regimen B→C | BM | 18 | ALLR3, high risk, clofarabine | Died of relapse (1.8 y) | |
| 11 | UKALL 2003 | 8 | F | 34 | Yes | Yes | >10 | Regimen A→C | BM | 50 | Allogenic transplant | Alive (3.8 y) | |
| 12 | UKALL 2003 | 16 | F | 68 | Yes | NK | >0.01 | Regimen B | No | Died in remission from infection (4 mo) | |||
| 13 | UKALL 2003 | 16 | F | 102 | No | Yes | >10 | NA | Primary refractory disease, treated on ALLR3 as high risk with clofarabine | Died of infection with active disease (2 mo) | |||
| 14 | UKALL2011 | 5 | F | 152 | Yes | Yes | >10 | Imatinib + regimen from EsPhALL trial | Allogeneic (MRD 0.003% before bone marrow transplant) | No | Died in remission 6 mo after stem cell transplant from undefined encephalopathy | ||
| 15 | UKALL2011 | 8 | F | 345 | Yes | Yes | > 10 | Regimen B→C + imatinib (at end of induction) | No | No | Alive in CR at 10 mo on maintenance therapy |
| Patient No. . | Trial . | Age (y) . | Sex . | WBC × 109/L . | CR . | Slow early responder* . | Postinduction MRD level (%) . | Treatment allocation† . | First remission transplant . | Relapse . | Months to relapse . | Relapse therapy . | Status . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | ALL 97 | 6 | F | 212 | Yes | Yes‡ | ND | Standard therapy | BM and CNS | 31 | Allogeneic transplant | Died of relapse (5 y) | |
| 2 | ALL 97/99 | 12 | F | 17 | Yes | Yes | ND | Regimen B→C | No | Alive (7.6 y) | |||
| 3 | ALL 97/99 | 12 | F | 5 | Yes | Yes | ND | Regimen B→C | BM | 39 | ALLR3, intermediate risk, mitoxantrone, MUD | Alive (7.4 y) | |
| 4 | UKALL 2003 | 13 | F | 60 | Yes | Yes | >10 | Regimen B→C | Allogeneic | BM and bone | 27 | NK | Alive (9.3 y) |
| 5 | UKALL 2003 | 4 | F | 49 | Yes | No | >0.1 | Regimen B→C | No | Alive (7.5 y) | |||
| 6 | UKALL 2003 | 11 | M | 70 | No | Yes | >10 | Regimen B→C | MUD | No | Alive (7.3 y) | ||
| 7 | UKALL 2003 | 19 | M | 8 | Yes | Yes | >10 | Regimen B | CNS | 40 | Allogeneic transplant | Alive (6.8 y) | |
| 8 | UKALL 2003 | 8 | M | 12 | Yes | No | >10 | Regimen A | Skin | 59 | ALLR3, standard risk, mitoxantrone | Alive (6 y) | |
| 9 | UKALL 2003 | 18 | F | 3 | Yes | Yes | >10 | Regimen B→C | Allogeneic | No | Alive (5.2 y) | ||
| 10 | UKALL 2003 | 14 | M | 26 | Yes | Yes | >10 | Regimen B→C | BM | 18 | ALLR3, high risk, clofarabine | Died of relapse (1.8 y) | |
| 11 | UKALL 2003 | 8 | F | 34 | Yes | Yes | >10 | Regimen A→C | BM | 50 | Allogenic transplant | Alive (3.8 y) | |
| 12 | UKALL 2003 | 16 | F | 68 | Yes | NK | >0.01 | Regimen B | No | Died in remission from infection (4 mo) | |||
| 13 | UKALL 2003 | 16 | F | 102 | No | Yes | >10 | NA | Primary refractory disease, treated on ALLR3 as high risk with clofarabine | Died of infection with active disease (2 mo) | |||
| 14 | UKALL2011 | 5 | F | 152 | Yes | Yes | >10 | Imatinib + regimen from EsPhALL trial | Allogeneic (MRD 0.003% before bone marrow transplant) | No | Died in remission 6 mo after stem cell transplant from undefined encephalopathy | ||
| 15 | UKALL2011 | 8 | F | 345 | Yes | Yes | > 10 | Regimen B→C + imatinib (at end of induction) | No | No | Alive in CR at 10 mo on maintenance therapy |
BM, bone marrow; CNS, central nervous system; F, female; M, male; MUD, matched unrelated donor; NA, not applicable; ND, not determined; NK, not known; WBC, white blood cell count.
Slow early responder, >25% blasts at first assessment (day 8 for regimen B and day 15 for regimen A).
Full details of the treatment for ALL 97/99 and UKALL 2003 regimens have been published.9,10 Briefly, in ALL 99 and UKALL 2003, patients were assigned to regimen A or B on the basis of whether they were National Cancer Institute standard (<10 years old and WBC <50 × 109/L) or high risk (≥10 years old or WBC >50 × 109/L), respectively, and were randomly assigned to regimen C on the basis of MRD stratification.
Day 8, 77% blasts.