Table 1.
Summary of patient characteristics and clinical results obtained with UniCAR-T-CD123 in patients with relapsed/refractory AML
| Characteristic | Patient number | ||
|---|---|---|---|
| 1 | 2 | 3 | |
| Age, y/sex | 54/male | 65/male | 80/male |
| AML characteristics (FAB-subtype, cytogenetics and molecular markers) | rr secondary AML, 5q- | rr AML (M2), RUNX, NRAS | rr AML (M0), ZRSR2 |
| Prior lines of treatment | Chemotherapy, haploidentical allogeneic HCT | Chemotherapy, unrelated allogeneic HCT, azacitidine | Chemotherapy, azacitidine, venetoclax |
| BM blasts prior to lymphodepletion (%) (CD123+ %) | 26 (80) | 20 (26) | 30 (80) |
| UniCAR-T dose | 100 × 106 | 250 × 106 | 250 × 106 |
| TM123 dose (mg/day) | 0.5 | 0.5 | 1.0 |
| Peak UniCAR-T-cell number/mL | 1860 | 408 | 1628 |
| Best response (% BM blasts) | PR (13) | CRi (0) | CRi (2) |
| CRS/NT | No CRS, no NT | Grade 1 CRS (2 d), no NT | Grade 1 CRS (2 d), no NT |
5q- RUNX, gene encoding for runt-related transcription factor; BM, bone marrow; CRi, complete remission with incomplete hematologic recovery; CRS, cytokine release syndrome; FAB, French-American-British; HCT, hematopoietic cell transplantation; NRAS, gene encoding for neuroblastoma RAS viral oncogene homolog; rr, relapsed/refractory; NT, neurotoxicity; PR, partial remission; ZRSR2, gene encoding for U2 small nuclear ribonucleoprotein auxiliary factor 35-kDa subunit-related protein 2.