Agustus 2021: Food and Drug Administration menyetujui idecabtagene vicleucel (Abecma, Bristol Myers Squibb) untuk pengobatan pasien dewasa dengan multiple myeloma yang kambuh atau sulit disembuhkan setelah empat atau lebih lini terapi sebelumnya, termasuk agen imunomodulator, inhibitor proteasome, dan anti-CD38 antibodi monoklonal. Ini adalah terapi gen berbasis sel pertama untuk multiple myeloma yang telah disetujui oleh FDA.
Idecabtagene vicleucel adalah perawatan sel T autologous chimeric antigen receptor (CAR) yang direkayasa secara genetik yang menargetkan antigen pematangan sel B (BCMA). Setiap dosis disesuaikan dengan sel T pasien sendiri, yang diambil, dimodifikasi secara genetik, dan kemudian diperkenalkan kembali ke pasien.
In a multicenter research, 127 patients with relapsed and refractory mieloma multipel 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 adalah 72 persen (95 persen CI: 62 persen, 81 persen), dengan tingkat CR 28 persen (95 persen CI 19 persen, 38 persen). Sebanyak 65 persen pasien yang mencapai CR tinggal di sana selama setidaknya satu tahun.
Peringatan 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 CAR-positif adalah kisaran dosis yang disarankan untuk idecabtagene vicleucel.
Referensi: https://www.fda.gov/
Periksa detailnya di sini:
