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Int J Hematol. 2015 Nov;102(5):602-10. doi: 10.1007/s12185-015-1869-y. Epub 2015 Sep 26.

Favorable outcome in non-infant children with MLL-AF4-positive acute lymphoblastic leukemia: a report from the Tokyo Children's Cancer Study Group.

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Division of Leukemia and Lymphoma, Children's Cancer Center, National Center for Child Health and Development, 2-10-1 Okura, Setagaya-ku, Tokyo, 157-8535, Japan.
Division of Transplantation and Cell Therapy, Children's Cancer Center, National Center for Child Health and Development, Tokyo, Japan.
Department of Pediatrics, Toho University, Tokyo, Japan.
Department of Pediatrics and Adolescent Medicine, Juntendo University School of Medicine, Tokyo, Japan.
Department of Pediatrics, Yamanashi University, Kofu, Japan.
Department of Pediatrics, University of Tsukuba, Tsukuba, Japan.
Department of Pediatric Hematology and Oncology Research, National Center for Child Health and Development, Tokyo, Japan.
Department of Hematology/Oncology, Saitama Children's Medical Center, Saitama, Japan.
Department of Pediatrics, St. Luke's International Hospital, Tokyo, Japan.


Unlike acute lymphoblastic leukemia (ALL) in infants, MLL gene rearrangement (MLL-r) is rare in ALL children (≥1 year old). The outcome and optimal treatment options for MLL-r ALL remain controversial. Among the 1827 children enrolled in the Tokyo Children's Cancer Study Group ALL studies L95-14, L99-15, L99-1502, L04-16, and L07-1602 (1995-2009), 25 MLL-r ALL patients (1.3 %) were identified. Their median age and leukocyte count at diagnosis was 2 years old (range 1-15 years) and 27,690/μL (range 1800-1,113,000/μL), respectively. All but one patient achieved complete remission (CR) after induction therapy, and 19 underwent allogeneic hematopoietic stem cell transplantation (HSCT) in first CR according to the protocol. The 5-year event-free survival (EFS) and overall survival (OS) rate were 60.0 % [standard error (SE), 9.7 %] and 64.0 % (SE 9.6 %), respectively. Notably, 9/12 cases with MLL-AF4-positive ALL are alive in continuous CR with a 75.0 % (SE 12.5 %) EFS rate. The causes of treatment failure were as follows: one induction failure, five relapses, and five transplant-related deaths. With intensive chemotherapy and allogeneic HSCT, favorable outcome of children (≥1 year old) with MLL-AF4-positive ALL was observed. However, considering the risk of acute and late toxicities associated with HSCT, its indication should be restricted.


Acute lymphoblastic leukemia; Children; Hematopoietic stem cell transplantation; MLL

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