Outcome of pediatric patients with acute myeloid leukemia (AML) and -5/5q- abnormalities from five pediatric AML treatment protocols: a report from the Children's Oncology Group.

Background: Abnormalities of chromosome 5q (-5/5q-) are associated with poor prognosis in adults with acute myeloid leukemia (AML). However, there are no large studies on outcomes of children with -5/5q- AML. To determine the disease correlates of this group, we retrospectively analyzed cytogenetic data from five studies of childhood AML.

Procedure: Data from patients whose cytogenetic clones included -5/5q-, with the exception of those with acute promyelocytic leukemia or Down syndrome, were included.

Results: Of the 2,240 patients with cytogenetic data available, 26 (1.2%) had -5 or 5q-. A significant number of these patients were age 11-21 (61.5%, P = 0.031) and had M0 morphology compared with patients without -5/5q- (24.0% vs. 2.8%, P < 0.001). Twenty-two of the 26 patients had a complete remission (CR) response to induction chemotherapy. The 5-year overall survival (OS) from the time of diagnosis for the -5/5q- patients was significantly lower than for patients without -5/5q- (27 ± 17% vs. 50 ± 2%, P = 0.027). Similarly, from induction CR, patients with -5/5q- had significantly worse disease free survival, OS and relapse risk than those without this abnormality (27 ± 19% vs. 46 ± 2%, P = 0.035, 32 ± 20% vs. 57 ± 2%, P = 0.025, 68 ± 21% vs. 45 ± 2%, P = 0.01, respectively).

Conclusions: Pediatric patients with AML and -5/5q- had a very poor outcome. These findings support the need for new or novel therapies for these patients.