ASH 2018 — Tisagenlecleucel yields durable response in pediatric ALL


  • Wayne Kuznar
  • Univadis
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Takeaway

  • An updated analysis of the ELIANA study shows that most pediatric and young adults with relapsed or refractory acute lymphocytic leukemia (ALL) remain in remission at 18 months without need for additional therapies after a single infusion of the CD19-targeted chimeric antigen receptor T-cell therapy tisagenlecleucel 

Why this matters

  • Patients had no other treatment options available at study entry.

Study design

  • Single-group, open-label phase 2 international study of 97 patients with a median of 3 prior lines of therapy.
  • Median age was 11 (range, 3-24) years; 61% had received a prior hematopoietic stem cell transplant.
  • Funding: Novartis.

Key results

  • Complete remission (CR) or CR with incomplete blood count recovery rate at 3 months (n=79) was 82% (95% CI, 72%-90%); CR rate was 62%..
  • At 18 months:
    • Relapse-free survival rate was 66% (95% CI, 52%-77%).
    • OS rate was 70% (95% CI, 58%-79%).
    • Median OS not reached.
  • 19 patients relapsed before receiving additional anticancer therapy, 8 underwent stem cell transplant while in remission, and 8 received additional anticancer therapy.
  • 77% of patients experienced grade 3-4 cytokine release syndrome (CRS), 39% of whom received tocilizumab for treatment (with or without other anticytokine therapies) and 48% of whom required treatment in the intensive care unit for a median of 7 days. All cases of CRS were reversible.

Limitation

  • Single-group, open-label study.