TY - JOUR
T1 - Tisagenlecleucel outcomes in relapsed/refractory extramedullary ALL
T2 - A Pediatric Real World CAR Consortium Report
AU - Fabrizio, Vanessa A.
AU - Phillips, Christine L.
AU - Lane, Adam
AU - Baggott, Christina
AU - Prabhu, Snehit
AU - Egeler, Emily
AU - Mavroukakis, Sharon
AU - Pacenta, Holly
AU - Rossoff, Jenna
AU - Stefanski, Heather E.
AU - Talano, Julie An
AU - Moskop, Amy
AU - Margossian, Steven P.
AU - Verneris, Michael R.
AU - Myers, Gary Douglas
AU - Karras, Nicole A.
AU - Brown, Patrick A.
AU - Qayed, Muna
AU - Hermiston, Michelle
AU - Satwani, Prakash
AU - Krupski, Christa
AU - Keating, Amy K.
AU - Wilcox, Rachel
AU - Rabik, Cara A.
AU - Chinnabhandar, Vasant
AU - Kunicki, Michael
AU - Yasemin Goksenin, A.
AU - Curran, Kevin J.
AU - Mackall, Crystal L.
AU - Laetsch, Theodore W.
AU - Schultz, Liora M.
N1 - Funding Information:
This work was supported by a St Baldrick's/StandUp2 Cancer Pediatric Dream Team Translational Cancer Research Grant (C.L.M.). StandUp2 Cancer is a program of the Entertainment Industry Foundation administered by the American Association for Cancer Research. C.L.M is a member of the Parker Institute for Cancer Immunotherapy, which supports the Stanford University Cancer Immunotherapy Program. The work was also supported by the Virginia and D.K. Ludwig Fund for Cancer Research.
Publisher Copyright:
© 2022 American Society of Hematology. All rights reserved.
PY - 2022/1/25
Y1 - 2022/1/25
N2 - Chimeric antigen receptor (CAR) T cells have transformed the therapeutic options for relapsed/refractory (R/R) B-cell acute lymphoblastic leukemia. Data for CAR therapy in extramedullary (EM) involvement are limited. Retrospective data were abstracted from the Pediatric Real World CAR Consortium (PRWCC) of 184 infused patients from 15 US institutions. Response (complete response) rate, overall survival (OS), relapse-free survival (RFS), and duration of B-cell aplasia (BCA) in patients referred for tisagenlecleucel with EM disease (both central nervous system (CNS)3 and non-CNS EM) were compared with bone marrow (BM) only. Patients with CNS disease were further stratified for comparison. Outcomes are reported on 55 patients with EM disease before CAR therapy (CNS3, n = 40; non- CNS EM, n = 15). The median age at infusion in the CNS cohort was 10 years (range, ,1-25 years), and in the non-CNS EM cohort it was 13 years (range, 2-26 years). In patients with CNS disease, 88% (35 of 40) achieved a complete response vs only 66% (10 of 15) with non- CNS EM disease. Patients with CNS disease (both with and without BM involvement) had 24-month OS outcomes comparable to those of non-CNS EM or BM only (P = .41). There was no difference in 12-month RFS between CNS, non-CNS EM, or BM-only patients (P = .92). No increased toxicity was seen with CNS or non-CNS EM disease (P = .3). Active CNS disease at time of infusion did not affect outcomes. Isolated CNS disease trended toward improved OS compared with combined CNS and BM (P = .12). R/R EM disease can be effectively treated with tisagenlecleucel; toxicity, relapse, and survival rates are comparable to those of patients with BM-only disease. Outcomes for isolated CNS relapse are encouraging.
AB - Chimeric antigen receptor (CAR) T cells have transformed the therapeutic options for relapsed/refractory (R/R) B-cell acute lymphoblastic leukemia. Data for CAR therapy in extramedullary (EM) involvement are limited. Retrospective data were abstracted from the Pediatric Real World CAR Consortium (PRWCC) of 184 infused patients from 15 US institutions. Response (complete response) rate, overall survival (OS), relapse-free survival (RFS), and duration of B-cell aplasia (BCA) in patients referred for tisagenlecleucel with EM disease (both central nervous system (CNS)3 and non-CNS EM) were compared with bone marrow (BM) only. Patients with CNS disease were further stratified for comparison. Outcomes are reported on 55 patients with EM disease before CAR therapy (CNS3, n = 40; non- CNS EM, n = 15). The median age at infusion in the CNS cohort was 10 years (range, ,1-25 years), and in the non-CNS EM cohort it was 13 years (range, 2-26 years). In patients with CNS disease, 88% (35 of 40) achieved a complete response vs only 66% (10 of 15) with non- CNS EM disease. Patients with CNS disease (both with and without BM involvement) had 24-month OS outcomes comparable to those of non-CNS EM or BM only (P = .41). There was no difference in 12-month RFS between CNS, non-CNS EM, or BM-only patients (P = .92). No increased toxicity was seen with CNS or non-CNS EM disease (P = .3). Active CNS disease at time of infusion did not affect outcomes. Isolated CNS disease trended toward improved OS compared with combined CNS and BM (P = .12). R/R EM disease can be effectively treated with tisagenlecleucel; toxicity, relapse, and survival rates are comparable to those of patients with BM-only disease. Outcomes for isolated CNS relapse are encouraging.
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U2 - 10.1182/bloodadvances.2021005564
DO - 10.1182/bloodadvances.2021005564
M3 - Article
C2 - 34794180
AN - SCOPUS:85123516789
VL - 6
SP - 600
EP - 610
JO - Blood advances
JF - Blood advances
SN - 2473-9529
IS - 2
ER -