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. 2023 Sep 15;142(21):1771–1783. doi: 10.1182/blood.2023020168

Table 2.

Selected studies evaluating the safety and efficacy of CAR T-cell therapy in SCNSL

Study Product N Response, % Survival, mo CRS, % Neurotoxicity, %
TRANSCEND105 Liso-cel 7 ORR, 50
CR, 50
NR Any grade, 29
Grade ≥3, 0
Any grade, 29
Grade ≥3, 29
NCT0460848793 Axi-cel 9 ORR, 78
CR, 67
mPFS, 11.5 Any grade, 88
Grade ≥3, 0
Any grade, 50
Grade ≥3, 37.5
Retrospective study106 Axi-cel 17§ ORR, 59
CR, NR
6-month EFS from infusion, 50% Any grade, 93
Grade ≥3, 13
Any grade, 87
Grade ≥3, 33
Retrospective study92 Tisa-cel 8 ORR, 50
CR, 25
NR Any grade, 87
Grade ≥3, 0
Any grade, 37
Grade ≥3, 0
Retrospective study107 Tisa-cel, 57%
Axi-cel, 43%
7 ORR, 86
CR, 86
mPFS, 2.8
mOS, 4.3
Any grade, 57
Grade ≥3, 14
Any grade, 43
Grade ≥3, 14
Retrospective study108 Axi-cel 5 ORR, 80
CR, 60
mPFS, 4.5
mOS, 5.2
Any grade, 40
Grade ≥3, 0
Any grade, 40
Grade ≥3, 40
Retrospective study109 Costimulatory domain
4-1BB, 80%
CD28, 20%
10 ORR, 70
CR, 20
mPFS (systemic), 3
mPFS (CNS), not reached
Any grade, 100
Grade ≥3, 10
Any grade, 60
Grade ≥3, 30

EFS, event-free survival; mPFS, median PFS; mOS, median OS; NR, not reported.

Six patients evaluable for response.

PCNSL, 67% and SCNSL, 33%.

Ongoing accrual and follow-up.

§

Fifteen patients evaluable for response.

One patient with stable disease.

Three patients with stable disease.