The outcomes of relapsed acute myeloid leukemia in children: Results
from the Japanese Pediatric Leukemia/Lymphoma Study Group (JPLSG)
AML-05R study
Abstract
Background: The prognosis of children with acute myeloid leukemia (AML)
has improved with the efficacy of hematopoietic cell transplantation as
a second-line therapy and improvements in supportive care following
anthracycline- and cytarabine-based chemotherapy; however, the outcomes
of children with relapsed AML still remain unsatisfactory. Procedure: In
order to identify prognostic factors and improve their prognosis, we
analyzed 111 patients who relapsed after treatment with the Japanese
Pediatric Leukemia/Lymphoma Study Group (JPLSG) AML-05 protocol and who
were registered in the retrospective JPLSG AML-05R study. Results: The
5-year overall survival rate was 36.1%. The major determinant of
survival was duration from the diagnosis to relapse. The mean duration
in the non-surviving group (10.1 ± 4.1 months) was shorter than that in
the surviving group (16.3 ± 8.3 months) (p<0.01). Moreover,
achieving a second complete remission (CR2) prior to hematopoietic cell
transplantation was associated with a good prognosis (p<0.01).
Etoposide, cytarabine and mitoxantrone (ECM)- or fludarabine, cytarabine
and granulocyte colony-stimulating factor (FLAG)-based regimens were
therefore recommended for reinduction therapy (p<0.01). A
genetic analysis also revealed the prognostic significance of FMS-like
tyrosine kinase 3 (FLT3)-internal tandem duplication as a poor
prognostic marker (p=0.04) and core binding factor-AML, t(8;21) and
inv(16), as good prognostic markers (p<0.01). Conclusions:
Achieving a CR2 prior to HCT is important in order to improve the
prognosis of relapsed pediatric AML. Recent molecular targeted
therapies, such as FLT3 inhibitors, may contribute to overcome their
prognoses. Larger prospective investigations are necessary to establish
individualized treatment strategies for patients with relapsed childhood
AML.