Table 1.
Baseline characteristics, treatment, and response of individual patients
| Pt (no.) |
Age/sex (year) |
Prior therapies (no.) |
BM blasts (%, by morphology) |
BM blasts (%, by flow cytometry) | EMDs | CD7+ in blasts (%) |
CAR T-cell source/Dose (105/kg) | Grade ≥ 3 CRS/onset (mos) |
Grade ≥ 3 GVHD/onset (mos) |
Grade ≥ 3 Infection/onset (mos) |
Response at day 30 | Follow-up time (mos)/remission status/live status |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| E001 | 12/M | 3 | < 5 | 1.35 | No | 99.98 | Prior-SCT/5 | No | No | No | CR | 9.0/Loss follow-up |
| E002 | 4/M | 3 | < 5 | 1.54 | No | 98.80 | Prior-SCT/5 | No | No | Yes/5.5 | CR | 5.5/Death of AE |
| E003 | 6/F | 3 | 9.00 | 18.61 | No | 100.00 | Prior-SCT/5 | Yes/< 1 | No | Yes/11–13.3 | CR | 28.8/Remission |
| E004 | 6/M | 4 | < 5 | 0.19 | No | 99.95 | Prior-SCT/10 | No | Yes/8.2 | Yes/12.3 | CR | 12.5/Death of AE |
| E005 | 10/M | 2 | 18.50 | 11.22 | Throat/Ileocecal/Inguen | 99.10 | New/10 | No | No | No | CR | 28.0/Remission |
| E006 | 7/M | 2 | 80.00 | 41.36 | No | 86.42 | New/10 | No | No | No | NR | 0.5/Loss follow-up |
| E007 | 18/M | 4 | 80.00 | 59.70 | No | 100.00 | Prior-SCT/5 | No | No | No | CR | 8.8/Relapse |
| E008 | 23/M | 4 | < 5 | 0 | Left neck | 95.00# | Prior-SCT/10 | Yes/< 1 | No | No | PR | 27.1/Remission |
| E009 | 19/M | 4 | < 5 | 3.86 | Kidney | 97.22 | Prior-SCT/10 | No | No | No | CR | 20.8/Relapse |
| E010 | 11/F | 4 | < 5 | 1.60 | No | 81.18 | Prior-SCT/10 | No | No | Yes/5.4–6.8 | CR | 6.8/Death of AE |
| E011 | 10/M | 2 | < 5 | 0.90 | No | 100.00 | New/10 | No | No | No | CR | 26.4//Relapse |
| E012 | 11/M | 2 | 56.50 | 39.29 | No | 100.00 | New/10 | No | No | No | CR | 17.0/Relapse |
| E013 | 17/M | 4 | 84.00 | 87.99 | CNS + Testicle | 100.00 | Prior-SCT/10 | No | No | No | Resp* | 1.8/Loss follow-up |
| E014 | 2/F | 2 | < 5 | 6.17 | No | 93.80 | New/10 | No | No | No | CR | 14.9/Relapse |
| E015 | 18/M | 3 | 6.00 | 5.77 | CNS, Optical nerves | 90.60 | New/10 | No | No | No | CR | 24.7/Remission |
| E016 | 17/M | 2 | < 5 | 1.17 | Mediastinum/Lymphonode | 100.00 | Prior-SCT/10 | No | No | Yes/8.7 | CR | 8.7/Death of AE |
| E017 | 43/F | 2 | 71.50 | 53.35 | Breast mass | 100.00 | New/10 | No | No | No | CR | 1.7/Death of AE |
| E018 | 33/M | 4 | 89.00 | 16.84 | No | 100.00 | Prior-SCT/10 | No | No | No | CR | 24.2/Remission |
| E019 | 5/F | 2 | < 5 | 72.40 | CNS | 100.00 | New/10 | No | No | No | CR | 24.1/Remission |
| E020 | 10/M | 3 | < 5 | 0 | CNS | 100.00 | Prior-SCT/10 | No | No | No | CR | 18.3/Relapse |
Bold indicates patient who received new donor-derived CAR T cells at 1 × 106 (± 30%)/kg, italics indicates patient who received prior-SCT donor-derived CAR T cells at 1 × 106 (± 30%)/kg, and bolditalics indicates patient who received prior-SCT donor-derived CAR T cells at 5 × 105 (± 30%)/kg
AE—adverse event; Pt—patient; no.—number; F—female; M—male; BM—bone marrow; EMDs—extramedullary diseases; CNS—central nervous system; IHC—immunohistochemistry; CR—complete remission; NR—non-response; CRS—cytokine release syndrome; GVHD—graft-versus-host disease; mos—months; SCT—stem cell transplantation
*This patient achieved objective response (MRD– in BM and CSF, and reduction in EMDs) until out of trial
#Determined by immunohistochemistry