What is the efficacy of chimeric antigen receptor (CAR) T-cell therapy for the treatment of acute lymphoblastic leukemia (ALL)?

Updated: May 04, 2018
  • Author: Sameh Gaballa, MD, MS; Chief Editor: Emmanuel C Besa, MD  more...
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ALL is the most common pediatric cancer, with approximately 3,100 cases diagnosed in children and adolescents younger than 20 years each year in the United States. Eighty to 85% of pediatric ALL cases originate in B cells  [5] .

Approximately 98% of children with ALL attain complete remission (CR) with standard treatment, and 85% of patients aged 1 to 18 years with newly diagnosed ALL treated with current regimens have long-term event-free survival.  [5]  However, relapsed or refractory ALL carries a poor prognosis: with current chemotherapy, fewer than 25% of patients achieve CR, and responses typically last only 4 to 9 weeks  [3] .

In patients with B-cell ALL, early studies with CAR T-cells targeting CD19 reported CR rates of 70%-90%, and remissions were often long-lasting. [3, 6, 7] . For example, in 2014, Maude et al reported a 90% CR rate in 30 children and adults with relapsed or refractory ALL. Nineteen patients had sustained remissions beyond 2 to 3 months, suggesting continued function of the infused cells. [3] .

Three patients in CR subsequently underwent allogeneic stem cell transplantation. In the patients who did not undergo transplantation, event-free survival at median follow-up of 6 months was 67%. In patients with relapse who received salvage therapy, overall survival at 6 months was 78%. [3]  However, longer follow-up is needed, since the median follow up for this study was short.

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