Table 1.
Patients’ disease characteristics prior to CD19hs CAR-T infusion.
| Patient No. | Age | Sex | Complex chromosome | Gene fusion | Previous CAR-T therapies | Outcomes and DOR (mon) after mCAR-T infusions | Bridging to HSCT | No. of treatment regimens before enrollment | |||
|---|---|---|---|---|---|---|---|---|---|---|---|
| Source of CAR scFv | Target | No. of infusions | Infusion dosage×10^6/kg | ||||||||
| 1 | 9 | M | N | E2A-HLF | murine | CD19 + CD22 | 1 | 0.3 + 0.3 | CR with MDR- for 1 | N | 6 |
| 2 | 14 | M | Y | E2A-HLF | murine | CD19 | 2 | 0.3 | CR with MRD- for 3.5 | N | 3 |
| CD19 + CD22 | 0.3 + 0.3 | NR | |||||||||
| 3 | 17 | M | N | BCR-ABL1 | murine | CD19 | 2 | 4 | CR with MRD- for 8 | N | 6 |
| CD19 | 0.3 | CR with MRD- for 1* | |||||||||
| 4$ | 14 | F | N | MLL/ITD | murine | CD19 | 1 | 1 | CR for 18** | Y | 4 |
| 5$$ | 20 | F | Y | MLL/ITD | murine | CD19 | 2 | 1 | NR | Y | 6 |
| CD19 | 1 | CR with MRD- for 12*** | |||||||||
| 6$$$ | 6 | F | Y | BCR-ABL1 | murine | CD19 + CD22 | 1 | 0.9 + 1 | CR with MRD- for 11 | Y | 6 |
| 7$$$$ | 19 | M | N | N | murine | CD19 | 2 | 0.06 | NR | N | 8 |
| CD19 | 0.5 | NR | |||||||||
| 8 | 13 | F | N | N | murine | CD19 | 2 | 0.06 | NR | Y | 6 |
| hu | CD19 | 0.64 | CR for 6**** | ||||||||
allo-HSCT, allogeneic hematopoietic stem cell transplantation; CD19hsCAR-T, chimeric antigen receptor T cells engineered with humanized selective CD19-specific scFv; mCAR-T, chimeric antigen receptor T cells engineered with murine-based scFv; mon, month(s); CR, complete remission; DOR, duration of remission, time spanning from CR as evaluated on day 30 post-infusion to either the time of relapse, death, loss to follow-up, or the present time when the manuscript was prepared in the case of ongoing sustained CR; MRD, minimal residual disease; hu, humanized; ITD: internal tandem duplication; F, female; M, male; N, no; NR, nonresponse; Y, yes.
$Patient 4 harbored gene mutations, including IKZF1 mutation, ERG (Δ3-9 positive), FANCD2 (C2080 G>A pD694N), NRAS (G13D) and JAK (I668F);
$$Patient 5 harbored gene mutations, including IKZF1 mutation, ERG (Δ3-9 positive) and NRAS (G13D);
$$$Patient 6 harbored gene mutation, including IKZF1 heterozygous deletion from Exo5-6, PAX5 heterozygous deletion from Exo 2-6, and Exo 8;
$$$$Patient 7 harbored gene mutation, including KRAS Q22K and ASXL1 T822Pfs*3;
*Patient 3 received the 2nd infusion of CD19mCAR-T as a preventive treatment;
**Patient 4 achieved CR for 18 mon with CD19mCAR-T infusion bridging to allo-HSCT;
***Patient 5 achieved CR for 12 mon with 2 consecutive CD19mCAR-T infusions, then bridging to allo-HSCT;
****Patient 8 did not respond to CD19mCAR-T, and then achieved CR for 6 mon with a humanized CD19CAR-T (from a different research group; CR with 0.11% MDR) bridging to haplo-HSCT (CR with MRD-).