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Disease Burden Affects Outcomes in Pediatric and Young Adult B-ALL After Commercial Tisagenlecleucel: A Pediatric Real-World CAR Consortium

<ѻý class="mpt-content-deck">– An ASCO Reading Room selection
Liora Schultz, MD; Christina Baggott, PhD; Snehit Prabhu, PhD; Holly Pacenta, MD; Christine Phillips, MD; Jenna Rossoff, MD; Heather Stefanski, MD, PhD; Julie-An Talano, MD; Amy Moskop, MD; Steven Margossian, MD, PhD; Michael Verneris, MD; Gary Douglas Myers, MD; Nicole Karras, MD; Patrick Brown, MD; Muna Qayed, MD, MSc; Michelle Hermiston, MD, PhD; Prakash Satwani, MD; Christa Krupski, DO, MPH; Amy Keating, MD; Rachel Wilcox, BS; Cara Rabik, MD, PhD; Vanessa Fabrizio, MD, MS; Rayne Rouce, MD; Vasant Chinnabhandar, MD; Michael Kunicki, BS; Valentin Barsan, MD; A. Yasemin Goksenin, MD, PhD; Yimei Li, PhD; Sharon Mavroukakis, MS; Emily Egeler, PhD; Kevin Curran, MD; Crystal Mackall, MD; and Theodore Laetsch, MD
Journal of Clinical Oncology

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Purpose

Tisagenlecleucel is a CD19-specific chimeric antigen receptor T-cell therapy, U.S. Food and Drug Administration–approved for children, adolescents, and young adults (CAYA) with relapsed and/or refractory (RR) B-cell acute lymphoblastic leukemia (B-ALL). The U.S. Food and Drug Administration registration for tisagenlecleucel was based on a complete response (CR) rate of 81%, 12-month overall survival (OS) of 76%, and event-free survival (EFS) of 50%. We report clinical outcomes and analyze covariates of outcomes after commercial tisagenlecleucel.

Methods

We conducted a retrospective, multi-institutional study of CAYA with RR B-ALL across 15 U.S. institutions, who underwent leukapheresis shipment to Novartis for commercial tisagenlecleucel. A total of 200 patients were included in an intent-to-treat response analysis, and 185 infused patients were analyzed for survival and toxicity.

Results

Intent-to-treat analysis demonstrates a 79% morphologic CR rate (95% CI 72-84%). The infused cohort had an 85% CR (95% CI 79-89%) and 12-month OS of 72% and EFS of 50%, with 335 days of median follow-up. Notably, 48% of patients had low-disease burden (< 5% bone marrow lymphoblasts, no CNS3, or other extramedullary disease), or undetectable disease, pretisagenlecleucel. Univariate and multivariate analyses associate high-disease burden (HB ≥ 5% bone marrow lymphoblasts, CNS3, or non-CNS extramedullary) with inferior outcomes, with a 12-month OS of 58% and EFS of 31% compared with low-disease burden (OS 85%, EFS 70%) and undetectable disease (OS 95%, EFS 72%; P<0.0001 for OS and EFS). Grade ≥ 3 cytokine release syndrome and neurotoxicity rates were 21% and 7% overall and 35% and 9% in patients with HB, respectively.

Conclusion

Commercial tisagenlecleucel in CAYA RR B-ALL demonstrates efficacy and tolerability. This first analysis of commercial tisagenlecleucel stratified by disease burden identifies HB preinfusion to associate with inferior OS and EFS and increased toxicity.

Read an interview about the study here and expert commentary about it here.

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Disease Burden Affects Outcomes in Pediatric and Young Adult B-ALL After Commercial Tisagenlecleucel: A Pediatric Real-World CAR Consortium

Primary Source

Journal of Clinical Oncolog

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ASCO Publications Corner