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Blood, 15 December 2005, Vol. 106, No. 13, pp. 4043-4049.
Prepublished online as a Blood First Edition Paper on August 18, 2005; DOI 10.1182/blood-2003-10-3446.
Previous Article | Next Article 
Submitted October 8, 2003
Accepted April 24, 2005
Clinical characteristics and outcome of children with down syndrome and acute lymphoblastic leukemia: a children's cancer group study
James A Whitlock, Harland N Sather, Paul Gaynon, Leslie L Robison, Robert J Wells, Michael Trigg, Nyla A Heerema, and Smita Bhatia*
Department of Pediatric Hematology/Oncology, Vanderbilt University Medical Center, Nashville, TN, USA
Children's Oncology Group, Arcadia, CA, USA
Children's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles, Los Angeles, CA, USA
Division of Epidemiology/Clinical Research, Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA
Division of Pediatrics, University of Texas M.D. Anderson Cancer Center, Houston, TX, USA
Division of Blood and Bone Marrow Transplantation, Alfred I. duPont Hospital for Children, Wilmington, DE, USA
Department of Cytogenetics, Children's Hospital of Columbus, Columbus, OH, USA
Division of Pediatrics, City of Hope Cancer Center, Duarte, CA, USA
* Corresponding author; email: SBhatia{at}coh.org.
We assessed the outcome of children with Down syndrome (DS) and acute lymphoblastic leukemia (ALL) receiving contemporary risk-based therapy by evaluating clinical and biologic features and outcome of children with ALL, with or without DS enrolled on Children's Cancer Group (CCG) protocols between 1983 and 1995. Comparison of characteristics of children with ALL with (ALL-DS, n=179) or without DS (ALL-NDS, n=8,268) showed no differences in initial white blood cell (WBC) count, central nervous system disease, and risk group. Children with ALL-DS did not present with unfavorable translocations, and were older than one year of age at diagnosis with ALL. Event-free (56% vs. 74%, p< 0.001) and disease-free survival (55% vs. 73%, p< 0.001) at 10 years was significantly lower in the standard-risk ALL-DS population compared with ALL-NDS, but not in high-risk ALL-DS population (EFS 62% vs. 59%, p=0.9; DFS 64% vs. 59%, p=0.9), and these differences persisted irrespective of treatment era [early era (1983 to 1989) vs. recent era (1989 to 1995)]. Multivariate analysis revealed that presence of DS demonstrated an independent significant adverse prognostic effect, for the standard-risk population, but not for the high-risk patients. These results suggest that intensification of therapy for patients with ALL-DS is needed to maintain outcome comparable to those of ALL-NDS patients.

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