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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Answer

Answer

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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