ETV6-RUNX1-positive childhood acute lymphoblastic leukemia: Improved outcome with contemporary therapy

D. Bhojwani, D. Pei, J. T. Sandlund, S. Jeha, R. C. Ribeiro, J. E. Rubnitz, S. C. Raimondi, S. Shurtleff, M. Onciu, C. Cheng, E. Coustan-Smith, W. P. Bowman, S. C. Howard, M. L. Metzger, H. Inaba, W. Leung, W. E. Evans, D. Campana, M. V. Relling, C. H. Pui

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83 Scopus citations


ETV6-RUNX1 fusion is the most common genetic aberration in childhood acute lymphoblastic leukemia (ALL). To evaluate whether outcomes for this drug-sensitive leukemia are improved by contemporary risk-directed therapy, we studied clinical features, response and adverse events of 168 children with newly diagnosed ETV6-RUNX1-positive ALL on St Jude Total Therapy studies XIIIA (N36), XIIIB (N38) and XV (N94). Results were compared with 494 ETV6-RUNX1-negative B-precursor ALL patients. ETV6-RUNX1 was associated with age 1-9 years, pre-treatment classification as low risk and lower levels of minimal residual disease (MRD) on day 19 of therapy (P0.001). Event-free survival (EFS) or overall survival (OS) did not differ between patients with or without ETV6-RUNX1 in Total XIIIA or XIIIB. By contrast, in Total XV, patients with ETV6-RUNX1 had significantly better EFS (P0.04; 5-year estimate, 96.82.4% versus 88.32.5%) and OS (P0.04; 98.91.4% versus 93.71.8%) than those without ETV6-RUNX1. Within the ETV6-RUNX1 group, the only significant prognostic factor associated with higher OS was the treatment protocol Total XV (versus XIIIA or XIIIB) (P0.01). Thus, the MRD-guided treatment schema including intensive asparaginase and high-dose methotrexate in the Total XV study produced significantly better outcomes than previous regimens and demonstrated that nearly all children with ETV6-RUNX1 ALL can be cured.

Original languageEnglish
Pages (from-to)265-270
Number of pages6
Issue number2
StatePublished - Feb 2012


  • ETV6-RUNX1
  • TEL-AML1
  • childhood acute lymphoblastic leukemia


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