• Yoshiyuki Kosaka, Katsuyoshi Koh, Naoko Kinukawa, Yoshihiro Wakazono, Keiichi Isoyama, Takanori Oda, Yasuhide Hayashi, Shigeru Ohta, Hiroshi Moritake, Megumi Oda, Yoshihisa Nagatoshi, Hisato Kigasawa, Yasushi Ishida, Akira Ohara, Ryouji Hanada, Masahiro Sako, Takeyuki Sato, Shuki Mizutani, Keizo Horibe, and Eiichi Ishii.
• Department of Hematology and Oncology, Hyogo Children's Hospital, Kobe, Japan.
• Blood. 2004 Dec 1; 104 (12): 3527-34.

AbstractForty-four infants with acute lymphoblastic leukemia (ALL) characterized by MLL gene rearrangements were treated on a protocol of intensive chemotherapy followed by hematopoietic stem cell transplantation (HSCT) between November 1998 and June 2002. The remission induction rate was 91.0%, and the 3-year overall survival and event-free survival (EFS) rates, with 95% confidence intervals, were 58.2% (43.5%-72.9%) and 43.6% (28.5%-58.7%), respectively. Univariate analysis of EFS by presenting features indicated a poorer outcome in patients younger than 6 months of age with high white blood cell counts (>/= 100 x 10(9)/L; EFS rate, 9.4% versus 55.1% for all others, P = .0036) and in those with central nervous system invasion (EFS rate, 10.0% versus 56.9% for all others, P = .0073). The 3-year posttransplantation EFS rate for the 29 patients who underwent HSCT in first remission was 64.4% (46.4%-82.4%). In this subgroup, only the timing of HSCT (first remission versus others) was a significant risk factor by multivariate analysis (P < .0001). These results suggest that early introduction of HSCT, possibly with a less toxic conditioning regimen, may improve the prognosis for infants with MLL(+) ALL. Identification of subgroups or patients who respond well to intensified chemotherapy alone should have a high priority in future investigations.

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