KYMRIAH (tisagenlecleucel), anti-CD19 CAR T

High clinical benefit in refractory B-cell acute lymphoblastic leukaemia, relapsed after transplantation, or after the second or later relapse in young adult patients up to the age of 25 years, and moderate clinical added value in the therapeutic strategy.

• KYMRIAH has been granted a marketing authorisation for the treatment of children and young adults up to the age of 25 years suffering from refractory B-cell ALL, relapsed after transplantation, or after the second or later relapse.

• CAR T cells are a gene therapy consisting of autologous T lymphocytes harvested by leukapheresis, then genetically modified ex vivo to express a chimeric receptor antigen targeting protein CD19 found on the B cell line. The re-injected anti-CD19 CAR T cells then multiply and activate in vivo after binding to target cells expressing CD19, thus inducing their apoptosis.

• High rates of complete remission have been achieved in two non-comparative studies (approximately 67% in the intention-to-treat population). During the last follow-up analysis, these remissions were maintained in approximately 40% of patients. KYMRIAH is associated with frequent adverse events whose severity can potentially require a stay in intensive care.

• Further data are awaited, considering the uncertainties concerning the efficacy, tolerance and complexity of the treatment process.

• The use of anti-CD19 CAR T is limited to a small number of specifically qualified centres.

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