Patient characteristics
| Patient no./ dose level . | Age at infusion/ sex . | Prior HCT . | Prior antigen-directed therapy . | Genetic alterations . | Cytogenetics . | Disease indication . | Before infusion∗ . | Max CRS/ NTX grade . | carHLH . | Response (BM; 4 wk after infusion) . | ||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| CNS status . | PET† . | Marrow blast/ MRD (%) . | ||||||||||
| 1/1 | 20.4/M | N | B | KMT2A-AFF1 | pseudodiploid | Relapse 3 | 2 | Pos | 98 (ND) | 1/1 | Y | NR |
| 2/1 | 18.5/F | N | N | SMU1-JAK2‡ | normal | Relapse 2 | 1 | ND | 10 (20.6) | 0/0 | N | MRDneg CR§ |
| 3/1 | 16.3/M | N | N | none identified | normal | Primary Refractory | 1 | ND | 1 (6.1) | 0/0 | N | MRDneg CR§ |
| 4/1 | 10.4/M | Y | B | IKZF1 truncation | hyperdiploid | Relapse 2 | 1 | Pos | 0 (0.058) | 0/1 | N | MRDneg CR§ |
| 5/1 | 15.4/F | Y | N | none identified | normal | Relapse 3 | 1 | Neg | 12 (56) | 1/0 | N | MRDneg CR§ |
| 6/1 | 1.8/F | Y | B/I | KMT2A-RELA | pseudodiploid | Relapse 2 | 1 | Pos | 0 (NGS 70) | 1/0 | N | MRDneg CR§ |
| 7/2 | 12.4/F | Y | B | JAK/STAT iAMP21‡ | hypodiploid/ pseudodiploid | Relapse 2 | 1 | Pos | 80 (43) | 0/0 | N | NR |
| 8/2 | 15.4/F | N | B | CRLF2-r‡ | hyperdiploid | Relapse 2 | 1 | ND | 72 (95.2) | 3/3 | N | MRDneg CR |
| 9/2 | 6.2/F | N | N | none identified | hyperdiploid | Relapse 2 | 2 | Neg | 78 (83) | 4/0 | Y | NR |
| 10/2 | 5.6/F | N | N | NUMA1-CSF1R‡ | ND | Relapse 2 | 1 | ND | 84 (59.2) | 1/0 | N | MRDneg CR§ |
| 11/2 | 21.8/M | N | N | none identified | normal | Relapse 2 | 1 | Pos | Aplastic (6.8) | 0/0 | N | MRDneg CR§ |
| 12/2 | 12.3/F | N | N | none identified | hyperdiploid | Relapse 1 (refractory) | 1 | Neg | 2 (0.07) | 0/0 | N | MRDneg CR |
| Patient no./ dose level . | Age at infusion/ sex . | Prior HCT . | Prior antigen-directed therapy . | Genetic alterations . | Cytogenetics . | Disease indication . | Before infusion∗ . | Max CRS/ NTX grade . | carHLH . | Response (BM; 4 wk after infusion) . | ||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| CNS status . | PET† . | Marrow blast/ MRD (%) . | ||||||||||
| 1/1 | 20.4/M | N | B | KMT2A-AFF1 | pseudodiploid | Relapse 3 | 2 | Pos | 98 (ND) | 1/1 | Y | NR |
| 2/1 | 18.5/F | N | N | SMU1-JAK2‡ | normal | Relapse 2 | 1 | ND | 10 (20.6) | 0/0 | N | MRDneg CR§ |
| 3/1 | 16.3/M | N | N | none identified | normal | Primary Refractory | 1 | ND | 1 (6.1) | 0/0 | N | MRDneg CR§ |
| 4/1 | 10.4/M | Y | B | IKZF1 truncation | hyperdiploid | Relapse 2 | 1 | Pos | 0 (0.058) | 0/1 | N | MRDneg CR§ |
| 5/1 | 15.4/F | Y | N | none identified | normal | Relapse 3 | 1 | Neg | 12 (56) | 1/0 | N | MRDneg CR§ |
| 6/1 | 1.8/F | Y | B/I | KMT2A-RELA | pseudodiploid | Relapse 2 | 1 | Pos | 0 (NGS 70) | 1/0 | N | MRDneg CR§ |
| 7/2 | 12.4/F | Y | B | JAK/STAT iAMP21‡ | hypodiploid/ pseudodiploid | Relapse 2 | 1 | Pos | 80 (43) | 0/0 | N | NR |
| 8/2 | 15.4/F | N | B | CRLF2-r‡ | hyperdiploid | Relapse 2 | 1 | ND | 72 (95.2) | 3/3 | N | MRDneg CR |
| 9/2 | 6.2/F | N | N | none identified | hyperdiploid | Relapse 2 | 2 | Neg | 78 (83) | 4/0 | Y | NR |
| 10/2 | 5.6/F | N | N | NUMA1-CSF1R‡ | ND | Relapse 2 | 1 | ND | 84 (59.2) | 1/0 | N | MRDneg CR§ |
| 11/2 | 21.8/M | N | N | none identified | normal | Relapse 2 | 1 | Pos | Aplastic (6.8) | 0/0 | N | MRDneg CR§ |
| 12/2 | 12.3/F | N | N | none identified | hyperdiploid | Relapse 1 (refractory) | 1 | Neg | 2 (0.07) | 0/0 | N | MRDneg CR |
B, blinatumomab; carHLH, CAR-associated hemophagocytic lymphohistiocytosis; CNS, central nervous system; F, female; HCT, hematopoietic cell transplant; I, inotuzumab; M, male; MRD, minimal residual disease (flow-based unless otherwise specified); MRDneg CR, MRD-negative complete response; N, no; ND, not done; Neg, negative; NGS, next-generation sequencing; NR, no response; PET, positron emission tomography; Pos, positive; Y, yes.
Data presented as patient number unless otherwise specified; dose level 1 = 1 × 106 CAR+ cells per kg; dose level 2 = 3 × 106 CAR+ cells per kg.
Disease status was assessed within 2 weeks of start of lymphodepleting chemotherapy and after any bridging therapy.
Positive PET defined as presence of hypermetabolic activity outside of BM and/or lymph nodes.
Genetic alteration categorized as Ph-like.
NGS testing was available and resulted as <10−5.