Characteristics and Clinical Outcome of 153 Children With ALL
| . | In Vitro PRD Resistance Assay . | P Value . | |
|---|---|---|---|
| Sensitive . | Resistant . | ||
| No. of patients | 108 | 45 | |
| Age in months, median (range) | 61 (4-190) | 70 (2-184) | .71 |
| Female/male ratio | 44/64 | 21/24 | .50 |
| WBC × 109/L, median (range) | 21.9 (1.7-519) | 37.2 (2.7-900) | .17 |
| BFM risk factor,* median (range) | 1.0 (0.3-1.9) | 1.1 (0.4-2.0) | .19 |
| FAB type | .19 | ||
| L1 | 84 | 38 | |
| L2 | 23 | 5 | |
| AUL† | 1 | 2 | |
| Immunophenotype | .007 | ||
| Pro-B | 3 | 3 | |
| Common | 55 | 22 | |
| Pre-B | 32 | 4 | |
| T | 18 | 16 | |
| DNA ploidy | .03‡ | ||
| Nonhyperdiploid | 85 | 36 | |
| Hyperdiploid | 17 | 1 | |
| Unknown | 6 | 8 | |
| Clinical response to PRD2-153 | .042-155 | ||
| Good | 97 | 34 | |
| *Blasts day 0 ≥ 1,000/μL | 88 | 26 | |
| *Blasts day 0 < 1,000/μL | 9 | 8 | |
| Poor | 6 | 6 | |
| Unknown | 5 | 5 | |
| Events | .002# | ||
| No complete remission | 0 | 1 | |
| Relapse | 24 | 18 | |
| Early death | 1 | 1 | |
| Toxic death | 3 | 0 | |
| None | 74 | 17 | |
| Lost to follow-up or otherwise not evaluable | 6 | 8 | |
| . | In Vitro PRD Resistance Assay . | P Value . | |
|---|---|---|---|
| Sensitive . | Resistant . | ||
| No. of patients | 108 | 45 | |
| Age in months, median (range) | 61 (4-190) | 70 (2-184) | .71 |
| Female/male ratio | 44/64 | 21/24 | .50 |
| WBC × 109/L, median (range) | 21.9 (1.7-519) | 37.2 (2.7-900) | .17 |
| BFM risk factor,* median (range) | 1.0 (0.3-1.9) | 1.1 (0.4-2.0) | .19 |
| FAB type | .19 | ||
| L1 | 84 | 38 | |
| L2 | 23 | 5 | |
| AUL† | 1 | 2 | |
| Immunophenotype | .007 | ||
| Pro-B | 3 | 3 | |
| Common | 55 | 22 | |
| Pre-B | 32 | 4 | |
| T | 18 | 16 | |
| DNA ploidy | .03‡ | ||
| Nonhyperdiploid | 85 | 36 | |
| Hyperdiploid | 17 | 1 | |
| Unknown | 6 | 8 | |
| Clinical response to PRD2-153 | .042-155 | ||
| Good | 97 | 34 | |
| *Blasts day 0 ≥ 1,000/μL | 88 | 26 | |
| *Blasts day 0 < 1,000/μL | 9 | 8 | |
| Poor | 6 | 6 | |
| Unknown | 5 | 5 | |
| Events | .002# | ||
| No complete remission | 0 | 1 | |
| Relapse | 24 | 18 | |
| Early death | 1 | 1 | |
| Toxic death | 3 | 0 | |
| None | 74 | 17 | |
| Lost to follow-up or otherwise not evaluable | 6 | 8 | |
Divided into two groups according to the extent of in vitro resistance to PRD: highly and intermediately sensitive (LC50 < 150 μg/mL) and resistant (LC50 ≥ 150 μg/mL).
A measure for the leukemic cell burden, based on peripheral leukemic cell count, and liver and spleen size.10
Acute undifferentiated leukemia.
Analysis excluding cases with unknown ploidy.
Determined after 1 week systemic PRD monotherapy (plus one intrathecal injection with methotrexate) by the number of blasts/μL peripheral blood (if < 1,000 = > good responder, if ≥ 1,000 = > poor responder).
Comparison of the good versus poor responders within potentially poor responders only (>1,000 blasts/μL peripheral blood at start of systemic PRD monotherapy).
#Comparison of yes versus no complete remission or relapse.