Tisagenlecleucel outcomes in relapsed/refractory extramedullary ALL: A Pediatric Real World CAR Consortium Report

Vanessa A. Fabrizio, Christine L. Phillips, Adam Lane, Christina Baggott, Snehit Prabhu, Emily Egeler, Sharon Mavroukakis, Holly Pacenta, Jenna Rossoff, Heather E. Stefanski, Julie An Talano, Amy Moskop, Steven P. Margossian, Michael R. Verneris, Gary Douglas Myers, Nicole A. Karras, Patrick A. Brown, Muna Qayed, Michelle Hermiston, Prakash SatwaniChrista Krupski, Amy K. Keating, Rachel Wilcox, Cara A. Rabik, Vasant Chinnabhandar, Michael Kunicki, A. Yasemin Goksenin, Kevin J. Curran, Crystal L. Mackall, Theodore W. Laetsch, Liora M. Schultz*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

1 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)600-610
Number of pages11
JournalBlood Advances
Issue number2
StatePublished - Jan 25 2022
Externally publishedYes

ASJC Scopus subject areas

  • Hematology


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