Mac 2022: Selepas empat atau lebih baris terapi terdahulu, termasuk perencat proteasome (PI), agen imunomodulator (IMiD), dan antibodi monoklonal anti-CD38, Pentadbiran Makanan dan Dadah telah meluluskan ciltacabtagene autoleucel (CARVYKTI, Janssen Biotech, Inc.) untuk rawatan pesakit dewasa dengan pelbagai myeloma yang berulang atau refraktori.
Ciltacabtagene autoleucel ialah rawatan terapi sel T CAR reseptor antigen chimeric autologous yang direka bentuk secara genetik 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.
CARTITUDE-1 (NCT03548207) was an open label, multicenter percubaan klinikal that looked at the safety and efficacy of ciltacabtagene autoleucel in 97 patients with relapsed or refractory pelbagai myeloma who had received at least three prior lines of therapy, including a PI, an IMiD, and an anti-CD38 monoclonal antibody, and who had disease progression on or after the last chemotherapy regimen Patients were given 0.51.0106 CAR-positive viable T cells per kg body weight of ciltacabtagene autoleucel. Efficacy was determined by an Independent Review committee utilising the International Myeloma Working Group Uniform Response Criteria for Multiple Myeloma to assess overall response rate (ORR) and duration of response (DOR). The ORR was 97.9% (95 percent confidence interval: 92.7 percent, 99.7%). The median duration of response (DOR) was 21.8 months (95 percent CI: 21.8, NE) among the 95 patients who responded, with a median follow-up period of 18 months.
The CARVYKTI label includes a boxed warning for sindrom pelepasan sitokin (CRS), hemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS), Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), Parkinsonism and Guillain-Barré syndrome and their complications, and prolonged and/or recurrent cytopenia, which can all be fatal or life-threatening. Pyrexia, cytokine release syndrome, hypogammaglobulinemia, musculoskeletal pain, fatigue, infections, diarrhoea, nausea, encephalopathy, headache, coagulopathy, constipation, and vomiting were the most prevalent side effects of ciltacabtagene autoleucel.
CARVYKTI mempunyai penilaian risiko dan pelan mitigasi yang memerlukan hospital dan klinik yang mengedarkan terapi diperakui khususnya untuk mengiktiraf dan menangani ketoksikan CRS dan sistem saraf. FDA meminta syarikat itu menjalankan kajian pemerhatian pasca pemasaran yang melibatkan pesakit yang dirawat dengan ciltacabtagene autoleucel untuk menilai keselamatan jangka panjang.
CARVYKTI diberikan pada dos 0.5-1.0106 sel T berdaya maju positif CAR setiap kg berat badan, dengan dos maksimum 1108 sel T berdaya maju positif CAR setiap infusi.
