How is high-risk pediatric acute lymphoblastic leukemia (ALL) treated?

Updated: Jan 02, 2019
  • Author: Vikramjit S Kanwar, MBBS, MBA, MRCP(UK), FAAP; Chief Editor: Jennifer Reikes Willert, MD  more...
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The optimal treatment for patients with very high-risk (VHR) acute lymphoblastic leukemia (ALL) has not been determined; [19] however, some centers recommend allogeneic hematopoietic stem cell transplantation (HSCT) soon after first remission (CR1) is achieved. For the subset of patients with BCR-ABL gene rearrangement, the addition of imatinib to intensified chemotherapy produced survival results equivalent to allogeneic HSCT. [20] In addition, the ongoing COG randomized trial AALL1131 is evaluating the use of the experimental agent clofarabine in conjunction with intensified chemotherapy for VHR ALL.

A review of 1,041 patients with ALL and induction failure showed this population to be highly heterogeneous in their clinical features. Patients with T-cell ALL appeared to have a better outcome with allogeneic HSCT, whereas for patients with B-cell ALL and either age younger than 6 years or high hyperdiploidy, the value of HSCT was less certain. [21] For patients without a matched family donor, HSCT from an unrelated donor would therefore no longer be a reasonable treatment option for that subset, although it may be for other patients with VHR ALL. Large, multi-institutional, controlled trials are needed to confirm this recommendation.

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