Table 1.
Patient characteristics
| UPN | Age | Gender | Disease | Extra-medullary disease prior CARTs | Diagnosis to CARTs [months] | # Prior tx lines | Prior alloSCT/donor/source | Prior blina | Bridging* | Disease status at LD |
|---|---|---|---|---|---|---|---|---|---|---|
| 1 | 32 | F | B-ALL | Yes (CNS) | 117 | 9 | Yes (2×)/2× MUD/2 × PBSCT | Yes | Yes | CSF: 7 blasts /µl; BM: <5% blasts, MRD - |
| 2 | 21 | M | Pre-B-ALL | No | 15 | 4 | Yes/MUD/PBSCT | Yes | No | Molecular relapse (BM MRD 0.3%) |
| 3 | 67 | F | B-ALL | No | 15 | 2 | No | No | No | Molecular relapse (BM MRD 0.3%) |
| 4 | 32 | F | B-ALL | No | 8 | 2 | Yes/MUD/PBSCT | No | No | 6% blasts in BM |
| 5 | 63 | M | Pre-B-ALL | No | 20 | 4 | Yes/haplo/BMT | Yes | No | 8% blasts in BM |
| 6 | 28 | F | B-ALL | Yes (bone) | 22 | 7 | Yes/MUD/PBSCT | No | No | PD (right epicondyle, humerus) |
| 7 | 67 | F | MPAL | No | 97 | 4 | Yes/MUD/PBSCT | No | Yes | Molecular relapse (BM MRD 0.018%) |
| 8 | 36 | F | Pre-B-ALL | Yes (left mamma, parotid gland, lymph nodes (axillary, mediastinal, parailiacal, inguinal) | 8 | 5 | No | Yes | Yes | 48% blasts in BM and PD of extramedullary disease |
| 9 | 45 | M | B-ALL | No | 25 | 6 | Yes/MUD/PBSCT | No | No | Molecular relapse (BM MRD 0.36%) |
| 10 | 47 | F | B-ALL | No | 18 | 4 | Yes/MUD/PBSCT | Yes | No | Molecular relapse (BM MRD 0.3%) |
| 11 | 37 | M | B-ALL | No | 52 | 5 | Yes/MUD/PBSCT | Yes | No | BM blasts 6% |
| 12 | 36 | M | B-ALL | Yes (kidney, liver, bone) | 28 | 4 | Yes/MMUD/PBSCT | Yes | No | PR of extramedullary manifestations, BM: <5% blasts |
| 13 | 32 | M | B-ALL | Yes (bone) | 117 | 4 | Yes/MUD/PBSCT | Yes | Yes | PD of extramedullary manifestations (10th rib, both femurs, both tibias, left Os ilium), BM: <5% blasts |
| 14§ | 47 | M | B-ALL | No | 41 | 4 | Yes/MMUD/PBSCT | Yes | Yes | Molecular relapse (BM MRD 0.3%)/death due to PD prior LD |
| 15§ | 65 | M | B-ALL | No | 5 | 3 | No | Yes | Yes | 8% blasts in BM/death due to sepsis prior LD |
ALL acute lymphoblastic leukemia, alloSCT allogeneic stem cell transplantation, B-ALL common B cell leukemia, BM bone marrow, BMT bone marrow transplant, CART chimeric antigen receptor T cells, CNS central nervous system, CR complete remission, CSF cerebrospinal fluid, F female, haplo haploidentical hematopoietic stem cell transplantation, LD lymphodepletion, M male, MPAL mixed-phenotype acute leukemia, MRD minimal residual disease, MMUD HLA-mismatched unrelated donor, MUD HLA-matched unrelated donor, PBSCT peripheral blood stem cell transplant, PR partial remission, pre-B-ALL precursor B cell acute lymphoblastic leukemia, PD progressive disease, SD stable disease, tx therapy, UPN unique patient number
*Treatment administered between leukapheresis and lymphodepletion
§Patients #14 and #15 did not receive HD-CAR-1 CARTs