KYMRIAH (tisagenlecleucel), anti-CD19 CAR T
Reason for request
High clinical benefit in the third-line or later treatment of refractory or recurrent diffuse large B-cell lymphoma and minor clinical added value in terms of efficacy compared to current management.
- KYMRIAH has been granted a marketing authorisation for the third-line or later treatment of refractory or recurrent diffuse large B-cell lymphoma (DLBCL).
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.
Precise quantification of the clinical effect was difficult, failing any comparative studies with usual management, even though direct comparison was possible.
In intention-to-treat (ITT) population, the data showed a complete response rate of circa 24.2%, along with an estimated 12-month survival rate of approximately 40%, with a median follow-up period limited to 7 months and adverse events of grade ≥3 in 90% of cases, occasionally requiring hospitalisation 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.
Improvement in actual benefit
Ce produit a fait l'objet d'un avis d'efficience rendu par la commission évaluation médico-économique et santé publique le 15 janvier 2019.