Table 1.
Characteristics of 17 patients with hepatic sinusoidal obstruction syndrome (SOS) reported as a severe adverse event during treatment for acute lymphoblastic leukemia (ALL) in comparison to the remaining patient population of trial AIEOP-BFM ALL 2000.
| Patients with reported hepatic SOS (n = 17) n (%) |
Patients without reported hepatic SOS (n = 3966) n (%) |
aP | |
|---|---|---|---|
| Gender | |||
| Male | 12 (70.6) | 2193 (55.3) | |
| Female | 5 (29.4) | 1773 (44.7) | 0.231 |
| Age at diagnosis (years) | |||
| <10 | 14 (82.4) | 2946 (74.3) | |
| ≥10 | 3 (17.6) | 1020 (25.7) | 0.584 |
| Initial WBCb (109/l) | |||
| <50 | 13 (76.5) | 3139 (79.2) | |
| ≥50 | 4 (23.5) | 825 (20.8) | 0.766 |
| n.a. | - | 2 (0.1) | |
| Immunophenotype | |||
| B-cell precursor | 17 (100) | 3305 (85.2) | |
| T-cell precursor | – | 555 (14.3) | |
| other | – | 19 (0.5) | 0.223 |
| n.a. | – | 87 (2.2) | |
| CNS diseasec | |||
| No | 15 (88.2) | 3584 (90.4) | |
| Yes | 1 (5.9) | 135 (3.4) | 0.448 |
| n.a. | 1 (5.9) | 247 (6.2) | |
| ETV6-RUNX1 rearrangement | |||
| Negative | 13 (76.5) | 2883 (72.7) | |
| Positive | 4 (23.5) | 839 (21.2) | 0.999 |
| n.a. | – | 244 (6.2) | |
| Prednisone responsed | |||
| Good | 16 (94.1) | 3539 (89.2) | |
| Poor | 1 (5.9) | 394 (9.9) | 0.999 |
| n.a. | – | 33 (0.8) | |
| Risk groupe | |||
| SR | 6 (35.3) | 1305 (32.9) | |
| MR | 8 (47.1) | 2050 (51.7) | |
| HR | 3 (17.6) | 609 (15.4) | |
| Other | – | 2 (0.1) | 0.845 |
| TPMT | |||
| wild-type | 8 (35.3) | 755 (19.0) | |
| Heterozygous | 9 (47.1) | 54 (1.4) | |
| Deficient | – | 4 (0.1) | <0.0001 |
| n.a. | – | 3153 (79.5) | |
aPχ2 or Fisher’s exact test.
bWBC white blood cell count.
cCNS positive: puncture nontraumatic, >5 WBC/μL cerebrospinal fluid with identifiable blasts
dGood: <1000 leukemic blood blasts/µl on treatment day 8, poor: ≥1000/µl.
eRisk group stratification included minimal residual disease (MRD) analysis and required two MRD targets with sensitivities of ≤1 × 10−4, SR patients were MRD-negative on treatment days 33 and 78, HR patients had residual disease of ≥5 × 10−4 on treatment day 78, all the remaining MRD results were stratified into the MR group, further HR criteria were prednisone poor response or ≥5% leukemic blasts in the bone marrow on day 33 or positivity for t(4;11) or its molecular equivalent (MLL-AF4 gene fusion); were stratified into the high-risk group independent of their MRD results.