What is the role of molecular targeted therapy in the treatment of pediatric acute lymphoblastic leukemia (ALL)?

Updated: Jan 03, 2019
  • Author: Vikramjit S Kanwar, MBBS, MBA, MRCP(UK), FAAP; Chief Editor: Jennifer Reikes Willert, MD  more...
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Answer

A drug targeted at the underlying molecular defect that is unique to certain leukemias can have potent and specific antileukemic activity while producing minimal toxicity to normal cells. [31] The best example of molecular targeted therapy is imatinib mesylate, a selective BCR-ABL tyrosine kinase inhibitor, that is standard front-line treatment for Ph-positive chronic myeloid leukemia (CML). Combination regimens with imatinib and conventional chemotherapy have shown efficacy in Ph-positive acute lymphoblastic leukemia. [32, 20]

Imatinib is approved for children newly diagnosed with Ph+ ALL. Its approval was based on a trial involving 92 patients in which children (1 year or older) and young adults were divided into 5 groups to receive different durations of imatinib therapy along with conventional chemotherapy. Among the 50 children receiving the longest duration of imatinib, the 4-year progression-free survival rate was 70%. Increasing duration of imatinib therapy was associated with lower overall mortality. [20]

The use of tyrosine kinase inhibitor or JAK2 inhibitor therapy for Ph-like ALL will be evaluated in future clinical trials.


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