%0 Journal Article
%A Schubert, Maria-Luisa
%A Schmitt, Anita
%A Hückelhoven-Krauss, Angela
%A Neuber, Brigitte
%A Kunz, Alexander
%A Waldhoff, Philip
%A Vonficht, Dominik
%A Yousefian, Schayan
%A Jopp-Saile, Lea
%A Wang, Lei
%A Korell, Felix
%A Keib, Anna
%A Michels, Birgit
%A Haas, Dominik
%A Sauer, Tim
%A Derigs, Patrick
%A Kulozik, Andreas
%A Kunz, Joachim
%A Pavel, Petra
%A Laier, Sascha
%A Wuchter, Patrick
%A Schmier, Johann
%A Bug, Gesine
%A Lang, Fabian
%A Gökbuget, Nicola
%A Casper, Jochen
%A Görner, Martin
%A Finke, Jürgen
%A Neubauer, Andreas
%A Ringhoffer, Mark
%A Wolleschak, Denise
%A Brüggemann, Monika
%A Haas, Simon
%A Ho, Anthony D
%A Müller-Tidow, Carsten
%A Dreger, Peter
%A Schmitt, Michael
%T Treatment of adult ALL patients with third-generation CD19-directed CAR T cells: results of a pivotal trial.
%J Journal of hematology & oncology
%V 16
%N 1
%@ 1756-8722
%C London
%I Biomed Central
%M DKFZ-2023-01426
%P 79
%D 2023
%Z DKFZ-ZMBH Alliance
%X Third-generation chimeric antigen receptor (CAR)-engineered T cells (CARTs) might improve clinical outcome of patients with B cell malignancies. This is the first report on a third-generation CART dose-escalating, phase-1/2 investigator-initiated trial treating adult patients with refractory and/or relapsed (r/r) acute lymphoblastic leukemia (ALL).Thirteen patients were treated with escalating doses of CD19-directed CARTs between 1 × 106 and 50 × 106 CARTs/m2. Leukapheresis, manufacturing and administration of CARTs were performed in-house.For all patients, CART manufacturing was feasible. None of the patients developed any grade of Immune effector cell-associated neurotoxicity syndrome (ICANS) or a higher-grade (≥ grade III) catokine release syndrome (CRS). CART expansion and long-term CART persistence were evident in the peripheral blood (PB) of evaluable patients. At end of study on day 90 after CARTs, ten patients were evaluable for response: Eight patients (80
%K Humans
%K Adult
%K Neurotoxicity Syndromes
%K Leukapheresis
%K Adaptor Proteins, Signal Transducing
%K Antigens, CD19: therapeutic use
%K Acute lymphoblastic leukemia (ALL) (Other)
%K CART-associated toxicities (Other)
%K CD39 (Other)
%K Cytokine release syndrome (CRS) (Other)
%K Cytopenia (Other)
%K Immune effector cell-associated neurotoxicity syndrome (ICANS) (Other)
%K Investigator-initiated trial (IIT) (Other)
%K Third-generation chimeric antigen receptor (CAR) T cells (Other)
%K cell-associated neurotoxicity (NLM Chemicals)
%K Adaptor Proteins, Signal Transducing (NLM Chemicals)
%K Antigens, CD19 (NLM Chemicals)
%F PUB:(DE-HGF)16
%9 Journal Article
%$ pmid:37481608
%2 pmc:PMC10363324
%R 10.1186/s13045-023-01470-0
%U https://inrepo02.dkfz.de/record/277709