title: Clinical characteristics and outcome of children with Down syndrome and acute lymphoblastic leukemia: a Children's Cancer Group study.
abstract: 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 in Children's Cancer Group (CCG) protocols between 1983 and 1995. Comparison of characteristics of children with ALL with (ALL-DS; n = 179) or without (ALL-NDS; n = 8268) DS 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 1 year of age at diagnosis with ALL. Event-free (56% vs 74%; P < .001) and disease-free (55% vs 73%; P < .001) survival 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 (event-free survival, 62% vs 59%; P = .9; disease-free survival, 64% vs 59%; P = .9), and these differences persisted regardless of treatment era (early era [1983-1989] vs recent era [1989-1995]). 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 with those of ALL-NDS patients.
authors.affiliation: Department of Pediatric Hematology/Oncology, Vanderbilt University Medical Center, Nashville, TN, USA.
authors.forename: James A
authors.initials: JA
authors.lastname: Whitlock
authors.forename: Harland N
authors.initials: HN
authors.lastname: Sather
authors.forename: Paul
authors.initials: P
authors.lastname: Gaynon
authors.forename: Leslie L
authors.initials: LL
authors.lastname: Robison
authors.forename: Robert J
authors.initials: RJ
authors.lastname: Wells
authors.forename: Michael
authors.initials: M
authors.lastname: Trigg
authors.forename: Nyla A
authors.initials: NA
authors.lastname: Heerema
authors.forename: Smita
authors.initials: S
authors.lastname: Bhatia
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D018572:Disease-Free Survival; D004314:Down Syndrome / Q000150:complications / Q000473:pathology* / Q000628:therapy*; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D054198:Precursor Cell Lymphoblastic Leukemia-Lymphoma / Q000150:complications / Q000473:pathology* / Q000628:therapy*; D012008:Recurrence; D012307:Risk Factors; D016896:Treatment Outcome