Table 1.
Patient, disease, and treatment characteristics
| CD19 (peds) | CD22 | BCMA | CD19 (adult) | GD2 | Total | ||
|---|---|---|---|---|---|---|---|
| NCT01593696 | NCT02315612 | NCT02215967 | NCT02659943 | NCT02107963 | |||
| Diseases treated | ALL (n = 50), NHL (n = 2) | ALL (n = 52), NHL (n = 1) | MM | NHL | OS (n = 11), NB (n = 2) |
||
| n | 52 | 53* | 24 | 20 | 13 | 162 | |
| Demographics | Median age, (range) years | 13 (4-30) | 17 (4-30) | 55 (43-66) | 58 (39-69) | 19 (10-28) | 19 (4-69) |
| Male, n (%) | 41 (78.8) | 34 (64.2) | 12 (50) | 12 (60) | 10 (76.9) | 109 (67.3) | |
| # patients with 1-2 lines of prior therapy, n (%) | 15 (28.8) | 2 (3.8) | 0 (0) | 7 (35) | 1 (7.7) | 25 (15.4) | |
| # patients with 3-5 lines of prior therapy, n (%) | 23 (44.2) | 23 (43.4) | 6 (25) | 9 (45) | 9 (69.2) | 70 (43.2) | |
| # patients with >5 lines of prior therapy, n (%) | 14 (26.9) | 28 (52.8) | 18 (75) | 4 (20) | 3 (23.1) | 67 (41.4) | |
| Prior allogeneic HSCT, n (%)# | 23 (44.2) | 38 (71.6) | 0 (0) | 0 (0) | 0 (0) | 61 (37.7) | |
| Prior autologous HSCT, n (%) | 0 | 0 | 20 (83.3) | 7 (35) | 2 (15.4) | 29 (17.9) | |
| Prior distinct CAR T-cell therapy, n (%) | 2 (3.8) | 31 (58.5) | 0 (0) | 0 (0) | 0 (0) | 33 (20.4) | |
| Disease status | % marrow involvement, median (range), n (%) | 30% (0-99) | 69 (0-95) | 15 (0-40) | 5 (0-40) | N/A | |
| IgG <400 prior to LD chemo, n (%) | 5 (9.6) | 16 (30.2) | 10 (41.7) | 6 (30) | 0 (0) | 37 (22.8) | |
| ANC <500 14 d prior to LD chemo, n (%) | 13 (25) | 20 (37.7) | 0 (0) | 0 (0) | 0 (0) | 33 (20.4) | |
| ALC <200 14 d prior to LD chemo, n (%) | 3 (5.8) | 10 (18.9) | 4 (16.7) | 0 (0) | 0 (0) | 17 (10.5) | |
| Median ANC on day 0, (range) | 1590 (<200-6620) | 900 (<200-9790) | 990 (<200-5230) | 2335 (600-5810) | 2620 (300-7550) | 1590 | |
| Median ALC on day 0, (range) | <200 (<200-1360) | <200 (<200-1250) | <200 (<200 – 970) | <200 (<200-300) | <200 (<200-660) | <200 | |
| Treatment characteristics | Low dose LD† chemotherapy, n (%) | 37 (71.2) | 53 (100) | 24 (100) | 20 (100) | 0 (0) | 134 (82.7) |
| High dose LD‡ chemotherapy, n (%) | 7 (13.5) | 0 (0) | 0 (0) | 0 (0) | 13 (100) | 28 (17.3) | |
| Any CRS, n (%) | 37 (71.2) | 47 (88.7) | 18 (75) | 17 (85) | 10 (76.9) | 129 (79.6) | |
| CAR T-cell course and outcome | CRS grade 1-2, n (%) | 28 (53.8) | 42 (79.2) | 12 (50) | 15 (75) | 10 (76.9) | 107 (66) |
| CRS ≥grade 3, n (%) | 9 (17.3) | 5 (9.4) | 6 (25) | 2 (10) | 0 (0) | 22 (13.6) | |
| ICU admission, n (%) | 22 (42.3) | 20 (37.7) | 12 (50) | 9 (45) | 0 (0) | 63 (13.6) | |
| Tocilizumab administered, n (%) | 7 (13.5) | 20 (37.7) | 5 (20.8) | 2 (10) | 0 (0) | 33 (20.4) | |
| Corticosteroids administered, n (%) | 3 (5.8) | 18 (33.9) | 4 (16.7) | 2 (10) | 0 (0) | 27 (16.7) | |
| Objective response rate at D30§, n (%) | 32 (61.5) | 38 (71.7) | 11 (45.8) | 13 (65) | 0 (0) | 94 (58) |
ALC, absolute lymphocyte count (reported as units of cells per mm3); ALL, acute lymphoblastic leukemia; ANC, absolute neutrophil count (reported as units of cells per mm3); CRS, cytokine release syndrome; HSCT, hematopoietic stem cell transplant; ICU, intensive care unit; LD chemo, lymphocyte depleting chemotherapy; MM, multiple myeloma; NB, neuroblastoma; NHL, non-Hodgkin lymphoma; OS, osteosarcoma; Peds, pediatric population.
X patients who were retreated after undergoing allogeneic stem cell transplant were treated as newly enrolled patients after transplant.
Standard dose lymphocyte depleting regimen was fludarabine 30 mg/m2 for D3 and cyclophosphamide 300 mg/m2 on day −3 to −5 prior to cell infusion.
High dose LD regimens were either etoposide 100 mg/m2 on days −5 to −1 and ifosfamide 1800 mg/m2 on days −5 to −1; or fludarabine 25 mg/m2 on days −5 to −1 and cytarabine 2000 mg/m2 on days −5 to −1 with filgrastim 5 μg/kg daily starting on day −6 until ANC >1000 for 2 days; or cyclosphosphamide 1800 mg/m2 on days −3 to −2.
Many CAR T-cell trials have response rates that improve over time; this ORR reflects only response rates at D30 and not the true ORR of the CAR T-cell trial.