Augusts 2021: Pārtikas un zāļu pārvalde apstiprināja idekabtagēna viklecelu (Abecma, Bristol Myers Squibb) pieaugušo pacientu ar recidivējošu vai refraktāru multiplo mielomu ārstēšanai pēc četrām vai vairākām iepriekšējām terapijas līnijām, ieskaitot imūnmodulējošu līdzekli, proteasomu inhibitoru un anti-CD38. monoklonālās antivielas. Šī ir pirmā uz šūnām balstīta gēnu terapija multiplās mielomas ārstēšanai, ko apstiprinājusi FDA.
Idecabtagene vicleucel ir ģenētiski izstrādāts autologo himērisko antigēnu receptoru (CAR) T-šūnu apstrāde, kuras mērķis ir B-šūnu nobriešanas antigēns (BCMA). Katra deva ir pielāgota pacienta paša T-šūnām, kuras tiek novāktas, ģenētiski modificētas un pēc tam atkal ievadītas pacientam.
In a multicenter research, 127 patients with relapsed and refractory multiplā mieloma who had undergone at least three prior lines of antimyeloma therapy were evaluated for safety and efficacy; 88 percent had received four or more prior lines of therapies. The efficacy of idecabtagene vicleucel at doses ranging from 300 to 460 x 106 CAR-positive T cells was studied in 100 individuals. The overall response rate (ORR), complete response (CR) rate, and duration of response (DOR) were calculated using the International Myeloma Working Group Uniform Response Criteria for Multiple Myeloma by an independent response committee.
ORR bija 72 procenti (95 procenti TI: 62 procenti, 81 procenti) ar 28 procentu CR līmeni (95 procenti TI 19 procenti, 38 procenti). Kopumā 65 procenti pacientu, kuri sasniedza CR, palika tajā vismaz gadu.
Ielikts brīdinājums par citokīnu atbrīvošanas sindroms (CRS), neurologic toxicities, hemophagocytic lymphohistiocytosis/macrophage activation syndrome, and persistent cytopenias is included on the idecabtagene vicleucel label. CRS, infections, exhaustion, musculoskeletal pain, and hypogammaglobulinemia are the most prevalent side effects of idecabtagene vicleucel.
Idecabtagene vicleucel has a risk evaluation and mitigation plan that requires healthcare facilities dispensing the medicine to be specially certified in recognising and managing DRS and nervous system toxicities. The FDA is ordering the company to conduct a post-marketing observational study involving patients treated with idecabtagene vicleucel in order to assess long-term safety.
300 līdz 460 106 CAR pozitīvas T šūnas ir ieteicamais idekabtagēna viklecela devu diapazons.
Atsauce: https://www.fda.gov/
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