Characteristics and Clinical Outcome of 156 Children With ALL
| . | Clinical PRD Response . | P Value . | |
|---|---|---|---|
| Good . | Poor . | ||
| No. of patients | 144 | 12 | |
| Age in months, median (range) | 61 (3-190) | 86 (2-163) | .57 |
| Female/male ratio | 64/80 | 3/9 | .19 |
| WBC × 109/L, median (range) | 19.2 (0.5-900) | 129.7 (43-746) | <.001 |
| BFM risk factor,* median (range) | 1.0 (0.3-2.0) | 1.3 (0.9-1.9) | .024 |
| FAB type | .60† | ||
| L1 | 117 | 9 | |
| L2 | 27 | 2 | |
| AUL‡ | 0 | 1 | |
| Immunophenotype | <.001 | ||
| Pro-B | 4 | 2 | |
| Common | 81 | 0 | |
| Pre-B | 38 | 0 | |
| T | 21 | 10 | |
| DNA ploidy | .45 | ||
| Nonhyperdiploid | 112 | 11 | |
| Hyperdiploid | 18 | 1 | |
| Not yet available | 14 | 0 | |
| LC50 values PRD, median (range) (μg/mL) | 1.35 (<0.06->2,000) | 130.9 (0.43->2,000) | .0061-153 |
| Follow-up, median (range) (in months) | 46 (17-81) | 65 (40-79) | .15 |
| Events | .081-155 | ||
| No complete remission | 0 | 1 | |
| Relapse | 38 | 5 | |
| Early death | 0 | 0 | |
| Toxic death | 2 | 1 | |
| No event | 97 | 5 | |
| Lost to follow-up or otherwise not evaluable | 7 | 0 | |
| . | Clinical PRD Response . | P Value . | |
|---|---|---|---|
| Good . | Poor . | ||
| No. of patients | 144 | 12 | |
| Age in months, median (range) | 61 (3-190) | 86 (2-163) | .57 |
| Female/male ratio | 64/80 | 3/9 | .19 |
| WBC × 109/L, median (range) | 19.2 (0.5-900) | 129.7 (43-746) | <.001 |
| BFM risk factor,* median (range) | 1.0 (0.3-2.0) | 1.3 (0.9-1.9) | .024 |
| FAB type | .60† | ||
| L1 | 117 | 9 | |
| L2 | 27 | 2 | |
| AUL‡ | 0 | 1 | |
| Immunophenotype | <.001 | ||
| Pro-B | 4 | 2 | |
| Common | 81 | 0 | |
| Pre-B | 38 | 0 | |
| T | 21 | 10 | |
| DNA ploidy | .45 | ||
| Nonhyperdiploid | 112 | 11 | |
| Hyperdiploid | 18 | 1 | |
| Not yet available | 14 | 0 | |
| LC50 values PRD, median (range) (μg/mL) | 1.35 (<0.06->2,000) | 130.9 (0.43->2,000) | .0061-153 |
| Follow-up, median (range) (in months) | 46 (17-81) | 65 (40-79) | .15 |
| Events | .081-155 | ||
| No complete remission | 0 | 1 | |
| Relapse | 38 | 5 | |
| Early death | 0 | 0 | |
| Toxic death | 2 | 1 | |
| No event | 97 | 5 | |
| Lost to follow-up or otherwise not evaluable | 7 | 0 | |
Divided into two groups according to their clinical response to a 1-week systemic PRD monotherapy: good responders (<1,000 blasts/μL PB at day 8) and poor responders (≥1,000 blasts/μL PB at day 8 of treatment).
A measure for the leukemic cell burden, based on peripheral leukemic cell count, and liver and spleen size.10
Comparison of FAB L1 versus no L1.
Acute undifferentiated leukemia.
The 17 patients with <1,000 blasts/μL blood at the start of treatment are excluded from this analysis to perform the most meaningful vitro-vivo correlation. However, including these patients still results in a significant 88-fold difference at P = .013.
Comparison of yes versus no complete remission or relapse.