Long-Term Clinical Outcomes of Tisagenlecleucel in Pediatric and Young Adult Patients With Relapsed/Refractory ALL.
Stephan A Grupp, Shannon L Maude, Susana Rives et al.
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In 30 seconds
This 5-year analysis of the global phase II ELIANA trial assessed long-term outcomes after a single tisagenlecleucel infusion in 79 children and young adults with relapsed or refractory B-cell acute lymphoblastic leukemia. With median follow-up of 79.4 months, estimated 5-year relapse-free survival among responders was 47.3% when stem cell transplantation was included in censoring for new anticancer therapy, and 51.0% when it was not. Median overall survival was not reached; estimated 5-year overall survival was 55.0% and 62.4%, respectively. No new or unexpected adverse events were reported.
Key findings
- Among 70 responders, estimated 5-year relapse-free survival was 47.3% with and 51.0% without inclusion of transplantation in censoring.
- Median overall survival was not reached; estimated 5-year overall survival was 55.0% with and 62.4% without inclusion of transplantation in censoring.
- Seventeen responders underwent postinfusion stem cell transplantation, including 14 who remained in complete remission at transplantation.
- No new or unexpected adverse events were reported.
Why it matters
The extended follow-up provides evidence that remission and survival can persist for years after tisagenlecleucel in some heavily pretreated children and young adults with relapsed or refractory B-cell ALL. Reporting outcomes under different censoring approaches, including for transplantation, helps clinicians interpret the durability of benefit while recognizing that subsequent treatment affects long-term estimates.
What to keep in mind
This is a phase II trial analysis of 79 patients; long-term estimates are affected by censoring for loss to follow-up, withdrawal, death, and subsequent anticancer therapy, including transplantation.