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3356 Efficacy of CAR-T Cells in Primary Central Nervous System Lymphomas: The French Experience of the National LOC Network

Program: Oral and Poster Abstracts
Session: 705. Cellular Immunotherapies: Late Phase and Commercially Available Therapies: Poster II
Hematology Disease Topics & Pathways:
Biological therapies, Lymphomas, non-Hodgkin lymphoma, Chimeric Antigen Receptor (CAR)-T Cell Therapies, Diseases, Therapies, Lymphoid Malignancies
Sunday, December 11, 2022, 6:00 PM-8:00 PM

Choquet Sylvain, MD1*, Magali Le Garff-Tavernier2*, Laetitia Souchet3*, Damien Roos Weil, MD, PhD4*, Veronique Morel, MD5*, Madalina Uzunov, MD6*, Carole Metz7*, Stephanie Nguyen Quoc, MDPC8*, Nathalie Jacque9*, Nicolas Gauthier, MD10*, Lise Willems, MD, PhD11*, Agathe Waultier, MD12*, Celia Salanoubat13*, Roch Houot, MD, PhD14*, Renata Ursu15*, Maryline Barrie16*, Guido Ahle, MD17*, Carole Soussain, MD18, Marion Alcantara, MD19* and Caroline Houillier, MD20*

1Clinical Hematology, La Pitie Salpetriere Hospital, APHP-Sorbonne Universite, Paris, France, France
2Sorbonne Université-INSERM UMRS 1138, AP-HP, Hopital Pitie-Salpetriere, Service d’Hématologie Biologique, Paris, France
3Pitie-Salpetriere Hospital - APHP Sorbonne Universite, Paris, France
4Hematology department, Sorbonne Université Hôpital Pitié Salpêtrière APHP, Paris, FRA
5Hematology Department, Pitié Salpétrière University Hospital, Paris, France
6Department of Hematology, University Hospital La Pitié SalpetrièRe, Paris, FRA
7Pharmacy, Groupe Hospitalier Pitié-salpêtrièRe, APHP, Paris, FRA
8Department of hematology, La Pitié University Hospital, Paris, France
9Service d'Hematologie Clinique, Hôpital Pitié Salpêtrière, APHP, Paris, France
10Service d’Hématologie, Hôpital Pitié-Salpêtrière, Assistance Publique-Hôpitaux de Paris, Paris, France
11Assistance Publique-Hôpitaux de Paris.Centre-Université Paris Cité, Hôpital Cochin, Department of Hematology, Paris, France
12Department of Hematology, CHU Nimes, Nimes, France
13CH Sud Francilien, Hematology, CORBEIL ESSONNES, FRA
14Department of Hematology, Rennes University Hospital, Rennes, France
15Assistance Publique -hopitaux De Paris, Paris, FRA
16neuro oncology, AP hopitaux de Marseille, Marseille, France
17Neurology Department, CH Colmar, Colmar, France
18Hematology Department, Rene Huguenin Hospital, Institut Curie, Saint-Cloud, France
19Institut Curie, Paris, France
20LOC network, La Pitie Salpetriere Hospital, Assistance Publique Hopitaux de Paris, Paris, France


Primary lymphomas of the central nervous system (PCNSL) have recently benefited from therapeutic progress but after the 2nd line the prognosis is particularly poor, with a survival of less than 6 months. CAR-T cells are of major contribution for systemic lymphomas, but the indication in PCNSL is prohibited in the United States, in Europe the prohibition is not specified. Since 2020, French centers, within the French national LOC network, have treated PCNSL in 3rd line and above. We present here the real-life results of the use of commercial CAR-T cells in PCNSL and compare them to the results of patients in the LOC network who did not benefit from them.


We retrospectively selected from the French LOC network database the adult immunocompetent PCNSL patients treated with CAR-T cells from the 3rd line of treatment. As control, we studied PCNSL patients from the LOC database treated with any treatment, at least in 3rd line and considered not eligible for an autologous stem cell transplantation (ASCT) or in relapse after ASCT.


17 PCNSL patients (median age : 68, median Karnofsky performans status (KPS) : 80, 9 women, 8 men) were treated with CAR-T cells (tisa-cel : N=13, axi-cel : N=4) between May 2020 and May 2022. They had previously received a median of 3 lines of treatment, including ASCT in 10/17 cases. 13 had a cerebral involvement (+/- eye and CSF) and 4 an isolated CSF involvement. All but one patient received a bridging therapy before CAR-T cells, resulting in complete response (CR) (N=1), partial response (PR) (N=6), stable disease (SD) (N=3), progressive disease (PD) (N=7). The median follow-up after CAR-T cells was 17.8 months. Best response was CR in 9 patients, PR in 4 patients and PD in 4 patients (overall response rate (ORR) of 76%). Median progression-free survival (PFS) was 7 months and median overall survival (OS) was 15.6 months. 15/17 patients experienced a CRS, including one grade III-IV, 9/17 an ICANS, including 3 grade III-IV, and 5/15 grade III-IV cytopenia lasting more than 28 days. In the control group (N=247), median age was 68 and median KPS 60. The efficacy endpoints were significantly worse in the control group compared to the CAR-T group: ORR of 41% (p=0.004), median PFS of 2.9 months (p=0.04) and median OS of 4.8 months (p=0.02).


CAR-T cells are effective in PCNSL, with responses and toxicity close to those found in systemic NHL. In comparison with LOC network data, this processing significantly improves PFS and OS. Data on a larger number of patients will make it possible to define prognostic factors for response and refine the indications.

Disclosures: Sylvain: Novartis: Consultancy, Honoraria; gilead: Consultancy, Honoraria. Le Garff-Tavernier: Abbvie: Consultancy, Honoraria; Janssen: Honoraria. Willems: Abbvie: Consultancy, Membership on an entity's Board of Directors or advisory committees; AstraZeneca: Consultancy, Membership on an entity's Board of Directors or advisory committees; Janssen: Consultancy, Membership on an entity's Board of Directors or advisory committees; Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees. Houot: Kite/Gilead, Novartis, Incyte, Janssen, MSD, Takeda, Roche: Honoraria; Kite/Gilead, Novartis, BMS/Gelgene, ADC THerapeutics, Incyte, Miltenyi: Membership on an entity's Board of Directors or advisory committees. Soussain: Gossamer Bio: Other: travel funding ; AstraZeneca: Research Funding; Hangzhou Henzen Pharmaceuticals: Research Funding.

*signifies non-member of ASH