Baseline characteristics and CART details
| . | All patients . | Responders* . | Nonresponders . |
|---|---|---|---|
| n = 14 . | n = 9 . | n = 5 . | |
| Age at CART, median (range), y | 0 (0-9) | 2 (0-9) | 1 (0-2) |
| KMT2Ar status | |||
| Yes | 12 | 8 | 4 |
| No | 1 | 0 | 1 |
| Unknown | 1 | 1 | 0 |
| Frontline therapy EOI response | |||
| MRD-negative CR | 5 | 3 | 2 |
| MRD-positive CR | 7 | 5 | 2 |
| >M1 | 1 | 1 | 0 |
| Not assessed | 1 | 0 | 1 |
| Prior immunotherapy and HSCT | |||
| Blinatumomab | 3 | 3 | 0 |
| Inotuzumab | 3 | 1 | 2 |
| HSCT | 4 | 2 | 2 |
| Disease category before CART | |||
| Primary refractory | 5 | 4 | 1 |
| First relapse† | 5 | 2 | 3 |
| Second or greater relapse | 4 | 3 | 1 |
| MRD status before CAR | |||
| MRD-negative CR | 3 | 3 | 0 |
| MRD-positive CR | 6 | 6 | 0 |
| >M1 | 5 | 0 | 5 |
| ALC at time of T-cell collection, median (range), cells/μL | 1610 (240-3330) | 1564 (240-5120) | 1692 (300-3330) |
| CART cell dose infused, median (range), (×106 CAR T cells/kg) | 2.29 (1.3-4.6) | 2.65 (1.6-4.6) | 1.72 (1.3-2.4) |
| CRS | |||
| Any grade | 11 | 6 | 5 |
| ≥Grade 3 | 3 | 1 | 2 |
| HSCT while in CART-mediated remission‡ | |||
| Yes | 1 | 1 | 0 |
| No | 13 | 8 | 5 |
| Disease status after CART | |||
| Refractory to CART | 4 | 0 | 4 |
| Remains in CR after CART | 7 | 7 | 0 |
| Relapsed after CART | 3§ | 2 | 1 |
| Myeloid transformation | |||
| Yes | 4 | 1 | 3 |
| No | 3 | 1 | 2 |
| Death | 4 | 0 | 4 |
| Time after CART at last follow-up, median (range), d | 231 (44-856) | 290 (137-856) | 85 (44-389) |
| . | All patients . | Responders* . | Nonresponders . |
|---|---|---|---|
| n = 14 . | n = 9 . | n = 5 . | |
| Age at CART, median (range), y | 0 (0-9) | 2 (0-9) | 1 (0-2) |
| KMT2Ar status | |||
| Yes | 12 | 8 | 4 |
| No | 1 | 0 | 1 |
| Unknown | 1 | 1 | 0 |
| Frontline therapy EOI response | |||
| MRD-negative CR | 5 | 3 | 2 |
| MRD-positive CR | 7 | 5 | 2 |
| >M1 | 1 | 1 | 0 |
| Not assessed | 1 | 0 | 1 |
| Prior immunotherapy and HSCT | |||
| Blinatumomab | 3 | 3 | 0 |
| Inotuzumab | 3 | 1 | 2 |
| HSCT | 4 | 2 | 2 |
| Disease category before CART | |||
| Primary refractory | 5 | 4 | 1 |
| First relapse† | 5 | 2 | 3 |
| Second or greater relapse | 4 | 3 | 1 |
| MRD status before CAR | |||
| MRD-negative CR | 3 | 3 | 0 |
| MRD-positive CR | 6 | 6 | 0 |
| >M1 | 5 | 0 | 5 |
| ALC at time of T-cell collection, median (range), cells/μL | 1610 (240-3330) | 1564 (240-5120) | 1692 (300-3330) |
| CART cell dose infused, median (range), (×106 CAR T cells/kg) | 2.29 (1.3-4.6) | 2.65 (1.6-4.6) | 1.72 (1.3-2.4) |
| CRS | |||
| Any grade | 11 | 6 | 5 |
| ≥Grade 3 | 3 | 1 | 2 |
| HSCT while in CART-mediated remission‡ | |||
| Yes | 1 | 1 | 0 |
| No | 13 | 8 | 5 |
| Disease status after CART | |||
| Refractory to CART | 4 | 0 | 4 |
| Remains in CR after CART | 7 | 7 | 0 |
| Relapsed after CART | 3§ | 2 | 1 |
| Myeloid transformation | |||
| Yes | 4 | 1 | 3 |
| No | 3 | 1 | 2 |
| Death | 4 | 0 | 4 |
| Time after CART at last follow-up, median (range), d | 231 (44-856) | 290 (137-856) | 85 (44-389) |
ALC, absolute lymphocyte count; EOI, end of induction.
Defined as MRD-negative CR at day 28 after CART.
First relapse: patients with first relapse that was refractory to salvage therapy (n = 3), patients with first relapse after HSCT (n = 2).
The indication for the patient who received HSCT while in CART-mediated remission included preemptive per discretion of treating physician.
All retained CD19+ status, extramedullary (skin, n = 1), marrow only (n = 1), marrow and central nervous system (n = 1).