What is the likely cause of treatment failure in 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

More than 80% of children with ALL now can be cured. [1] However, the cause of treatment failure in the remaining 20% of patients is largely unknown, although several clues have emerged from GWAS. Poor outcome has been correlated with alteration of IKZF1, which encodes the lymphoid transcription factor IKAROS, [37] and Janus kinase mutations have been associated with a high risk of treatment failure. [38]

Because of the diverse nature of the disease, use of risk-directed therapy for all patients on the basis of molecular and pharmacogenetic characterization of the leukemic cells at the time of diagnosis is favored, and the COG is looking at a clinical trial to evaluate the use of tyrosine kinase inhibitors for Ph-like ALL.


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