Long-term outcome of 6-month maintenance chemotherapy for acute lymphoblastic leukemia in children

In the treatment of childhood acute lymphoblastic leukemia (ALL), excess shortening of maintenance therapy resulted in high relapse rate, as shown by our previous trial, TCCSG L92-13, in which maintenance therapy was terminated at 1 year from initiation of treatment. In this study, we aimed to confirm the long-term outcome of L92-13, and to identify who can or cannot be cured by shorter duration of maintenance therapy. To obtain sentinel cytogenetics information that had been missed before, we performed genetic analysis with genomic microarray and target intron-capture sequencing from diagnostic bone marrow smear. Disease-free survival (DFS) at 10 years from the end of therapy was 66.0 +/- 2.8%. Females (n= 138) had better DFS (74.6 +/- 3.7%) than males (n= 142, 57.5 +/- 4.2%, P= 0.002). Patients with TCF3-PBX1 (n= 11) and ETV6-RUNX1 (n= 16) had excellent DFS (90.9 +/- 8.7% and 93.8 +/- 6.1%, respectively), whereas high hyperdiploidy (n= 23) was the most unfavorable subgroup, with 56.6 +/- 10.3% of DFS. Short duration of therapy can cure more than half of pediatric ALL, especially females, TCF3-PBX1 and ETV6-RUNX1. Our retrospective observations suggest a gender/karyotype inhomogeneity on the impact of brief therapy.