Table 1.
Baseline characteristics from CAR T-cell therapy of 185 patients treated across 15 Pediatric Real-World CAR Consortium (PRWCC) centres, stratified by CAR indication.
| Tisagenlecleucel indication | Refractory |
1st Relapse |
≥2nd Relapse |
p-value |
|---|---|---|---|---|
| (N = 30) | (N = 67) | (N = 87) | ||
| Baseline patient characteristics | ||||
| Age at Infusion (years) | ||||
| Median (Range) IQR | 13 (3–24) 10–18 | 10 (<1–26) 5–17 | 13 (1–25) 9–18 | 0.1197 |
| Age at diagnosis | ||||
| Median (Range) IQR | 12.5 (2–23) 9–18 | 7 (<1–25) 3–14 | 7 (<1–22) 3–13 | 0.0043 |
| Sex | ||||
| Male | 17 (57%) | 36 (54%) | 57 (66%) | 0.3169 |
| Female | 13 (43%) | 31 (46%) | 30 (34%) | |
| Race/Ethnicity | ||||
| Non-hispanic white | 15 (50%) | 35 (51%) | 40 (46%) | 0.1913 |
| Hispanic | 15 (50%) | 25 (37%) | 29 (33%) | |
| Black/African American | 0 | 0 | 7 (8%) | |
| Asian | 0 | 2 (3%) | 5 (6%) | |
| Other | 0 | 5 (7%) | 6 (7%) | |
| NCCN cytogenetic risk classification | ||||
| High risk | 21 (70%) | 29 (43%) | 14 (16%) | <0.0001 |
| Intermediate risk | 6 (20%) | 15 (22%) | 28 (32%) | |
| Low Risk | 2 (7%) | 8 (12%) | 15 (17%) | |
| Unknown | 1 (3%) | 15 (22%) | 30 (34%) | |
| Disease burden (4 unknown) | ||||
| High Burden (≥5% bone marrow lymphoblasts, CNS3, non-CNS extramedullary disease) | 11 (37%) | 38 (57%) | 45 (52%) | 0.2629 |
| Low burden (<5% bone marrow lymphoblasts) | 10 (33%) | 16 (24%) | 15 (17%) | |
| Undetectable Disease | 9 (30%) | 12 (18%) | 24 (28%) | |
| Prior HSCT | ||||
| Yes | 0 | 10 (15%) | 38 (44%) | <0.0001 |
| Prior CD19-targeted therapy | ||||
| Yes | 3 (10%) | 10 (15%) | 25 (29%) | 0.0371 |