Λίστα αντικειμένων
The FDA approved tisagenlecleucel (Kymriah) for certain paediatric and young adult patients with B-cell acute lymphoblastic leukaemia (ALL). The cell-based gene therapy is approved in the United States for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse.Tisagenlecleucel is a genetically-modified autologous T-cell immunotherapy. Each dose of tisagenlecleucel is a customised treatment created using an individual patient’s own T-cells. The T-cells are collected and sent to a manufacturing centre where they are genetically modified to include a new gene that contains a specific protein -- chimeric antigen receptor (CAR) -- that directs the T-cells to target and kill leukaemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.The safety and efficacy of tisagenlecleucel were demonstrated in a multicentre clinical trial of 63 paediatric and young adult patients with relapsed or refractory B-cell precursor ALL. The overall remission rate within 3 months of treatment was 83%.Treatment with tisagenlecleucel has the potential to cause severe side effects. It carries a boxed warning for cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of CAR T-cells causing high fever and flu-like symptoms, and for neurological events. Both CRS and neurological events can be life-threatening.The FDA today also expanded the approval of tocilizumab (Actemra) to treat CAR T-cell-induced severe or life-threatening CRS in patients aged 2 years or older. In clinical trials in patients treated with CAR-T cells, 69% of patients had complete resolution of CRS within 2 weeks following 1 or 2 doses of tocilizumab. The FDA granted tisagenlecleucel Fast Track, Priority Review, and Breakthrough Therapy designations. The FDA granted approval of tisagenlecleucel to Novartis Pharmaceuticals Corp. To further evaluate the long-term safety, Novartis is also required to conduct a post-marketing observational study involving patients treated with tisagenlecleucel.
Presenter:" Spyridonidis
PArticipants: all medical staff