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Myelodysplastic and Myeloproliferative Disorders of Childhood: A Study of 167 Patients

Authors :
Susan K. Atwater
Peter G. Steinherz
Kevin Shannon
Jorge A. Ortega
Vivian Weinberg
Margaret Masterson
Jean E. Sanders
Jeffrey H. Davis
Beverly J. Lange
Sandra Luna-Fineman
Source :
Blood. 93:459-466
Publication Year :
1999
Publisher :
American Society of Hematology, 1999.

Abstract

Myelodysplastic syndromes (MDS) and myeloproliferative syndromes (MPS) of childhood are a heterogeneous group of clonal disorders of hematopoiesis with overlapping clinical features and inconsistent nomenclature. Although a number of genetic conditions have been associated with MDS and MPS, the overall contribution of inherited predispositions is uncertain. We report a retrospective study examining clinical features, genetic associations, and outcomes in 167 children with MDS and MPS. Of these patients, 48 had an associated constitutional disorder. One hundred one patients had adult-type myelodysplastic syndrome (A-MDS), 60 had juvenile myelomonocytic leukemia (JMML), and 6 infants with Down syndrome had a transient myeloproliferative syndrome (TMS). JMML was characterized by young age at onset and prominent hepatosplenomegaly, whereas patients with A-MDS were older and had little or no organomegaly. The most common cytogenetic abnormalities were monosomy 7 or del(7q) (53 cases); this was common both in patients with JMML and those with A-MDS. Leukemic transformation was observed in 32% of patients, usually within 2 years of diagnosis. Survival was 25% at 16 years. Favorable prognostic features at diagnosis included age less than 2 years and a hemoglobin F level of less than 10%. Older patients tended to present with an adult-type MDS that is accommodated within the French-American-British system. In contrast, infants and young children typically developed unique disorders with overlapping features of MDS and MPS. Although the type and intensity of therapy varied markedly in this study, the overall outcome was poor except in patients with TMS.

Details

ISSN :
15280020 and 00064971
Volume :
93
Database :
OpenAIRE
Journal :
Blood
Accession number :
edsair.doi...........9e721474cacf5676281dd06813aa95cd
Full Text :
https://doi.org/10.1182/blood.v93.2.459