Augustus 2021: Die Food and Drug Administration het idecabtagene vicleucel (Abecma, Bristol Myers Squibb) goedgekeur vir die behandeling van volwasse pasiënte met terugval of refraktêre veelvuldige myeloom na vier of meer vorige reëls van terapie, insluitend 'n immunomodulerende middel, 'n proteasoom inhibeerder en 'n anti-CD38 monoklonale teenliggaampies. Dit is die eerste selgebaseerde geenterapie vir veelvuldige myeloom wat deur die FDA goedgekeur is.
Idecabtagene vicleucel is 'n geneties gemanipuleerde outoloë chimeriese antigeenreseptor (CAR) T-sel behandeling wat die B-sel rypwordingsantigeen (BCMA) teiken. Elke dosis word aangepas vir die pasiënt se eie T-selle, wat geoes word, geneties gemodifiseer word en dan weer in die pasiënt ingebring word.
In a multicenter research, 127 patients with relapsed and refractory veelvuldige myeloom 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.
Die ORR was 72 persent (95 persent CI: 62 persent, 81 persent), met 'n 28 persent CR-koers (95 persent CI 19 persent, 38 persent). Altesaam 65 persent van pasiënte wat CR bereik het, het vir ten minste 'n jaar daarin gebly.
N doos waarskuwing vir sitokienvrystellingsindroom (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 tot 460 106 CAR-positiewe T-selle is die voorgestelde dosisreeks vir idecabtagene vicleucel.
Verwysing: https://www.fda.gov/
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