Register number | Population (treated/enrolled) and age | Prior HSCT | Costimulatory domain and transduction method | Lymphodepletion | Subset and dose of infused CAR T cells | ORRb and MRD− CR rate | Long-term survival | Patient number and time for consolidative allo-HSCT | Reasons for not receiving allo-HSCT | transplant-related toxicities | Relapse rate, % (n) | Relapse rate after consolidative HSCT | Relapse rate without consolidative HSCT |
---|---|---|---|---|---|---|---|---|---|---|---|---|---|
20/20 5 to 27 | 35.0% (7/20) | CD28, retroviral | Flu/Cy | unselected, 1 × 106/kg to 3 × 106/kg | 70.0% (14/20), 60.0% (12/20) | OS: 51.6% after 9.7 m, EFS: 78.8% after 4.8 m | Patient number: 83.3% (10/12) MRD− CR patients, time: 45 to 82 (median 51) days | ineligible | no unexpected toxicity | 16.7% (2/12) MRD− CR patients | 0.0%, (0/10) | 100.0% (2/2) | |
51/51 2 to 68 | / | 4-1BB, lentiviral | Flu/Cy | unselected, 0.05 × 105/kg to 14 × 105/kg | 91.8% (45/49), 87.8% (43/49) | OS: /, LFS: 81.3% (6 m) after HSCT | Patient number: 60.0% (27/45) CR/CRi patients, time: 35 to 293 (median 84) days | / | / | 24.4% (11/45) CR/CRi patients | 7.4% (2/27) | 50.0% (9/18) | |
NCT01626495 & NCT01029366 [20] | 30/30a 5 to 60 | 60% (18/30) | 4-1BB, lentiviral | multiple | unselected, 0.76 × 106/kg to 20.6 × 106/kg | 90% (27/30), 73.3% (22/30) | OS: 78% (6 m), EFS: 67% (6 m) | Patient number: 11.1% (3/27) CR patients, time: / | lack of suitable donor, prior HSCT, family choice | / | 25.9% (7/27) CR patients | 0.0% (0/3) | 29.2% (7/24) |
NCT02435849 [4] | 75/92 3 to 23 | 61.3% (46/75) | 4-1BB, lentiviral | Flu/Cy or Ara-C/VP16 | unselected, 0.2 × 106/kg to 5.4 × 106/kg | 81.3% (61/75), 81.3% (61/75) | OS: 90% (6 m), 76% (12 m), EFS: 73% (6 m), 50% (12 m), LFS: 80% (6 m), 59% (12 m) | Patient number: 13.1% (8/61) CR/CRi patients, time: within 6 months | / | hepatobiliary disorders | 36.1% (22/61) CR/CRi patients | 0.0% (0/4), 4 others with unknown status | 41.5% (22/53) |
NCT01044069 [1] | 53/83 ≥18 | 35.8% (19/53) | CD28, retroviral | Flu/Cy or Cy | unselected, 1 × 106 or 3 × 106/kg | 83.0% (44/53), 66.7% (32/48) | OS: median 12.9 m, EFS: median 6.1 m | Patient number: 50.0% (16/32) MRD− CR patients, time: 44 to 312 (median 74) days | / | 6 died from transplant-related toxic effects | 50.0% (16/32) MRD− CR patients | 37.5% (6/16) | 62.5% (10/16) |
NCT02028455 [2] | 43/45 1.3–25.3 | 65.1% (28/43) | 4-1BB, lentiviral | Flu/Cy or Cy | CD4+ and CD8+ CAR T cells (1:1), 0.5 × 106/kg to 10 × 106/kg | 93% (40/43), 93% (40/43) | OS: 69.5% (12 m), EFS: 50.8% (12 m) | Patient number: 28% (11/40) MRD− CR patients, time: / | / | / | 45.0% (18/40) MRD− CR patients | 18.1% (2/11) | 55.2% (16/29) |
20/21, 5 to 48 | 50.0% (10/20) | CD28, retroviral | Flu/Cy | unselected, 1 × 106/kg | 90.0% (18/20), 78.6% (11/14) | OS: 90% (12 m), EFS: 73% (12 m) | Patient number: 77.8% (14/18) CR patients, time: median 68 days | / | / | 22.2% (4/18) CR patients | 14.3% (2/14) | 50.0% (2/4) | |
53/59, 20 to 76 | 43.4% (23/53) | 4-1BB, lentiviral | Flu/Cy or Cy | CD4+ and CD8+ CAR T cells (1:1), 2 × 105/kg or 2 × 106/kg | 84.9% (45/53), 84.9% (45/53) | (for MRD− CR patients) OS: median 20.0 m, EFS: median 7.6 m | Patient number: 40.0% (18/45) MRD− CR patients, time: 44 to 138 (median 70) days | age, the history of a prior transplant, patient preference, comorbidities | invasive fungal infection (aspergillus pneumonia), idiopathic acute respiratory distress syndrome, hepatic failure secondary to adenovirus infection, GVHD | 48.9% (22/45) MRD− CR patients | 16.7% (3/18) | 70.4% (19/27) | |
NCT02965092 & NCT03366350 [28] | 58/60 ≤70 | 5.2% (3/58) | 4-1BB, lentiviral | Flu/Cy | CD4+ and CD8+ CAR T cells (1:1), 0.89 × 106/kg to 4.01 × 106/kg | 87.9% (51/58), 81.0% (47/58) | OS: median 16.1 m, EFS: median 7.3 m | Patient number: 44.7% (21/47) MRD− CR patients, time: 33 to 89 (median 44) days | previous allo-HSCT, contraindications, lack of suitable donors, personal reasons | GVHD, pulmonary infection | 38.3% (18/47) MRD− CR patients | 9.5% (2/21) | 61.5% (16/26) |
110/115, 2 to 61 | 14.5% (16/110) | 4-1BB or CD28, lentiviral | Flu/Cy | unselected, 1 × 105/kg to 10 × 105/kg | 92.7% (102/110), 87.3% (96/110) | OS: 63.9% (12 m), EFS: 57.9% (12 m) | Patient number: 73.5% (75/102) CR patients, time: 36 to 120 (median 63) days | personal preference, performance status, financial issues, and donor availability | GVHD | 22.5% (23/102) CR patients | 13.3% (10/75) | 48.1% (13/27) |