2021 Ogos: Pentadbiran Makanan dan Dadah meluluskan idecabtagene vicleucel (Abecma, Bristol Myers Squibb) untuk rawatan pesakit dewasa dengan myeloma berganda yang berulang atau refraktori selepas empat atau lebih baris terapi terdahulu, termasuk agen imunomodulator, perencat proteasome dan anti-CD38 antibodi monoklonal. Ini adalah terapi gen berasaskan sel pertama untuk pelbagai myeloma yang telah diluluskan oleh FDA.
Idecabtagene vicleucel ialah rawatan sel T reseptor antigen chimeric autologous (CAR) autologous yang menyasarkan antigen pematangan sel B (BCMA). Setiap dos disesuaikan dengan sel-T pesakit sendiri, yang dituai, diubah suai secara genetik, dan kemudian dimasukkan semula ke dalam pesakit.
In a multicenter research, 127 patients with relapsed and refractory pelbagai myeloma 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 ialah 72 peratus (95 peratus CI: 62 peratus, 81 peratus), dengan kadar CR 28 peratus (95 peratus CI 19 peratus, 38 peratus ). Sejumlah 65 peratus pesakit yang mendapat CR tinggal di dalamnya selama sekurang-kurangnya setahun.
Amaran kotak untuk sindrom pelepasan sitokin (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 CRS 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 hingga 460 106 sel T positif CAR ialah julat dos yang dicadangkan untuk idecabtagene vicleucel.
Rujukan: https://www.fda.gov/
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