The Real Life Accessibility to CAR T-Cell Therapy: Current Experience in the Only Active Center in Italy

BACKGROUND. Axicabtagene ciloleucel and tisagenlecleucel have been approved by FDA and EMA for the treatment of relapsed/refractory diffuse large B-cell and mediastinal Lymphoma (NHL) patients (pts). Selection of pts who can benefit the most from these novel treatments with a low risk of life-threatening toxicities is currently a matter of discussion and outside clinical trials the selection of pts is up to clinicians of the CAR T-cell team in several countries. However, based on the results and follow up of clinical trials and the US reports about real life treatment with CAR T-cells, it is emerging that an expert clinical assessment and application of some inclusion criteria could optimize the success of therapy and minimize the severity of adverse events. AIMS.We are conducting a single center prospective observational trial to evaluate the accessibility and feasibility of CAR T-cells treatment among the population of NHL pts potentially eligible to this therapy. METHODS. Since September 2018 we have prospectively registered all pts referred at our center for CART-cells eligibility evaluation either for the enrollment in clinical trials or in the contest of the expanded access program (EAP) open for enrollment since February 2019 at our Institution. We have recorded clinical data including disease characteristics, comorbidities, history and present disease status at imaging. Patients were evaluated and screened for inclusion/exclusion criteria of the CAR T-cells program available at that moment and planned for treatment. RESULTS. Fifty-four pts with relapsed or refractory NHL potentially eligible to treatment according to EMA were recorded in 10 months. Median age was 48 yrs (range, 20 - 70). Thirty-nine pts were affected by DLBCL and 15 by PMBCL, all pts were refractory or relapsed to at least two chemotherapy regimens, median number of previous therapy was 3. Overall, among the 54 pts referred to our center only 7 pts (13%) have been enrolled in CAR T-cells programs (4 pts treated, 2 pts are waiting for infusion, one is in screening for a protocol) whereas other 11 (20%) have been considered eligible for CART-cells treatment but are still waiting for availability of treatment-slots. On the contrary 36 pts were considered not eligible. Seventeen pts (31%) were excluded after the first CAR T cell team visit because of rapidly progressive disease, or ECOG >1 or lymphoma mass larger than 20 cm and 7 pts (13%) were excluded for comorbidities. Nine pts requ