OBJECTIVEThere are very limited data on childhood acute promyelocytic leukemia (APL), especially childhood APL treated with arsenic trioxide (As(2)O(3)).

METHODBetween January 1999 and December 2007, 76 children (< 18 years) with newly diagnosed APL were included.

RESULTSSix cases failed to achieve hematological complete remission (CR) due to early death. Three groups of patients were identified according to the different induction regimen. Forty-four patients were given all-trans retinoic acid (ATRA) alone (group1, G1), 7 patients were given As(2)O(3) alone (group 2, G2), 19 patients (group 3, G3) were treated with combination of ATRA and As(2)O(3). The CR rate of G1 was 100%. The overall CR rate of G2 + G3 was 100%. Five children underwent hematologic relapse and 2 developed molecular relapse. The 5-year cumulative incidence of relapse was 13.8%, whereas event-free (EFS), disease-free (DFS) and overall survival rates were 79.5%, 86.3% and 90.5%, respectively. The 5-year estimate of EFS and DFS between G1 and G2 + G3 had no significant difference.

CONCLUSIONAs(2)O(3) is an effective and well tolerable therapy for children with APL and it may be used in those who not only cannot bear side effects of ATRA but also the newly diagnosed and relapsed APL.