Table 3.
Long-term outcomes of patients with relapsed and/or refractory multiple myeloma receiving BCMA-targeted CAR T cells
| Study and year of publication (n) | CAR design, name and trial phase | Median follow-up (range) | ORR and ≥CRR | PFS | DOR |
|---|---|---|---|---|---|
| Zhao et al. 2022 (74)59 | Two BCMA-binding domains, CD8α H/T domain, 4-1BB co-stimulatory domain; LCAR-B38Ma,b; multicentre phase I | 48 months | ORR: 65/74 (88%); ≥CRR: 54/74 (73%) | Median PFS: 18 months | Median DOR: 23 months |
| Martin et al. 2023 (97)21 | Two BCMA-binding domains, a CD8α H/T domain, 4-1BB co-stimulatory domainb,117; ciltacabtagene autoleucel; multicentre phase Ib/II | 28 months | ORR: 95/97 (98%); ≥CRR: 80/97 (83%) | PFS: 55% at 27 months | Median DOR: NE (range 23 months to NE) |
| Mikkilineni et al. 2021 (25)62 | Fully human scFv, CD8α H/T domain, 4-1BB co-stimulatory domainc; FHVH33-CD8BBZ | NR (median PFS of 78 weeks) | ORR: 23/25 (92%); ≥CRR: 12/25 (48%) | Median PFS: 18 months | Median DOR: NR |
| Munshi et al. 2021 (128)22 | Mouse scFv, CD8α H/T domain, 4-1BB co-stimulatory domainb,20,116; idecabtagene vicleucel; multicentre phase II | 13 months (0.2–21 months) | ORR: 94/128 (73%); ≥CRR: 42/128 (33%) | Median PFS: 8.8 months | Median DOR: 11 months in all patients and 19 months in those with CR or better |
| Wang et al. 2021 (18)60 | Fully human scFv, CD8α H/T domain, 4-1BB co-stimulatory domainb,60; CT103A; single-centre phase I | 13 months | ORR: 18/18 (100%); ≥CRR: 13/18 (72%) | 1-year PFS: 58% | Median DOR: 325 days for all patients and 412 days for patients with CR or better |
| Li et al. 2021 (30)61 | Mouse scFv, CD8α hinge, CD28 co-stimulatory domainb,61; anti-BCMA CAR T cells; phase I | 13 months | ORR: 27/30 (90%); ≥CRR: 13/30 (43%) | Median PFS: 5.2 months | Median DOR: 148 days for responding patients (range 16–625 days) |
Studies meeting the following criteria were included in this table: median follow-up duration ≥1 year and >15 patients on study in addition to including most of the above data in the manuscript. Studies are listed in order of longest duration of follow-up. Studies targeting B cell maturation antigen (BCMA) in addition to other antigens and retrospective or real-world studies were not included. Chimeric antigen receptor (CAR) name is included if the CAR construct was developed into a commercial product; otherwise, the construct name is listed. ≥CRR, complete response rate or better; CR, complete response; DOR, duration of response; H/T, hinge and transmembrane; NE, not estimable; NR, not reported; ORR, overall response rate; PFS, progression-free survival; scFv, single-chain variable fragment. aLCAR-B38M is the same CAR as used in ciltacabtagene autoleucel. bThese studies used a lentiviral vector for construct delivery. cThis study used a gammaretroviral vector for construct delivery.