title: Incidence and treatment of potentially lethal diseases in transient leukemia of Down syndrome: Pediatric Oncology Group Study.
abstract: Transient leukemia (TL or transient myeloproliferative disorder) occurs in approximately 10% of newborn infants with Down syndrome. The disorder is characterized by the presence of megakaryoblasts in the peripheral blood; most cases resolve spontaneously within the first 3 months of life, and the child is well thereafter. However, there are cases in which a severe, potentially lethal form of disease develops, manifesting as hepatic fibrosis or cardiopulmonary failure. Hitherto, the incidence of these severe forms of the disease has not been reported. A prospective study of TL was conducted by the Pediatric Oncology Group (POG Study 9481) in which 48 children with TL were identified. Life-threatening disease occurred in nine patients (19%); seven had hepatic fibrosis and two had cardiopulmonary failure. Five children died of the disease within the first 3 months of life, none of whom received antileukemic therapy. One patient died on day 31 after receiving minimal therapy within 1 day of death. Three children received low-dose cytosine arabinoside (Ara-C) (0.4-1.5 mg/kg every 12 hours for 5 or 7 days). In all these patients, the disease resolved. It is concluded that potentially lethal disease is relatively common in TL, and the available evidence suggests that these diseases are responsive to low-dose Ara-C therapy.
authors.affiliation: Division of Hematology/Oncology, The Hospital for Sick Children, Toronto, Ontario, Canada.
authors.forename: Fawaz
authors.initials: F
authors.lastname: Al-Kasim
authors.forename: John J
authors.initials: JJ
authors.lastname: Doyle
authors.forename: Gita V
authors.initials: GV
authors.lastname: Massey
authors.forename: Howard J
authors.initials: HJ
authors.lastname: Weinstein
authors.forename: Alvin
authors.initials: A
authors.lastname: Zipursky
mesh_terms: D002648:Child; D018450:Disease Progression; D004906:Erythrocyte Count; D017809:Fatal Outcome; D005260:Female; D006331:Heart Diseases / Q000097:blood / Q000150:complications* / Q000503:physiopathology; D006801:Humans; D015994:Incidence; D007231:Infant, Newborn; D007938:Leukemia / Q000453:epidemiology* / Q000401:mortality / Q000628:therapy; D007958:Leukocyte Count; D008171:Lung Diseases / Q000097:blood / Q000150:complications* / Q000503:physiopathology; D008297:Male; D008533:Megakaryocytes; D009504:Neutrophils; D010490:Pericardial Effusion / Q000097:blood / Q000150:complications / Q000503:physiopathology*