Presentation

DESCAR-T, National Registry for Patients with Hematological malignancies, Eligible for CAR T-Cell Therapy

DESCAR-T stands for “Dispositif d’Enregistrement et de Suivi des CAR-T” in French (Registry system to Register and Follow-up CAR T-Cell Therapy).

Why DESCAR-T ?

Among recent medical innovations, CAR T-cells represent a new therapeutic option that brings hope in the fight against some types of blood cancers. The Marketing Authorizations (MAs) of the first 2 CAR-Ts (KYMRIAH and YESCARTA) have been delivered based on phase II study data, with a limited follow-up duration at the time of registration. A Temporary Authorization of Use (ATU) was delivered in May 2021, ahead of the MA, for a 3rd CAR-T (ABECMA) in myeloma. Additional efficacy and safety data are thus needed to improve knowledge on CAR T-cells and have been requested by the French Health Authorities. Pharmaceutical compagnies and scientific experts have set up a collaboration to develop an independent registry for all CAR‐T therapies on the market. This registry currently includes the pharmaceutical companies Gilead, Novartis and BMS as well as the expert groups LYSA, IFM, GRAALL, SFCE and SFGM-TC. Other collaborations will be set up as MAs are delivered for other CAR-T therapies with treatment indications of hematological malignancies.

This registry will allow the follow-up of patients who have received CAR T-cells. Its aim is to better characterize the short- and long-term efficacy and safety profile of these treatments used in real-life setting.

Real-life studies are a valuable complement to standard clinical studies:

  • They allow for the collection of information on routine care practices in a patient population closer to the general patient population.
  • For example, they are key to validating the efficacy and safety of anti-cancer drugs in patient populations that would not be included in standard clinical trials.

What is the purpose of DESCAR-T?

The DESCAR-T registry is the result of a cooperation between several groups, namely GRAALL, IFM, LYSA, SFCE and SFGM-TC. It aims to follow approximately 1500 patients, adults and children, eligible to a treatment by CAR T-cells and diagnosed with certain types of hematological malignancies, across France.

Real-life data will be collected in the DESCAR-T registry on patients eligible to CAR-T over a period of 15 years, as requested by health authorities, allowing to draw a complete picture of the CAR-T landscape in France. The study is conducted in the RNIPH (Research Not Involving the Human Person) framework and uses health data already contained in the patient medical records, generated during the patient routine care pathway. It will be possible to study the correlations between clinical data and biological, imaging, quality-of-life data… to improve the medical understanding of CAR-T.

LYSARC is the sponsor of this research.

The analysis of these data will allow the evaluation of the efficacy and safety of CAR T-cells in real life, the description of the population eligible for this type of treatment and the place of these innovative treatments in the current therapeutic strategies.