Myelodysplastic syndromes in children. A critical review of the clinical manifestations and management.

摘要: The FAB group has defined myelodysplasia in adults but direct application of this categorization to children been controversial. Consequently, outline the natural history disease better we have retrospectively analysed case reports and series published English between 1982 1996. This study also included with juvenile chronic myelomonocytic leukaemia (JCML) monosomy 7 (Mo7). 340 patients were described 27 publications. mean presentation age was 5.91 (SD 5.04) years, 34.9% female. Constitutional alterations 68 (20%) where refractory anemia (RA) RA excess blasts (RAEB) predominated associated a significantly longer survival. Among all progression higher forms MDS noted 61 (18%). Cytogenetic anomalies detected 59% 227 children, 67 it Mo7. Amid those Mo7, clinical laboratory characteristics as well survival, closely followed their type. Of treatment options described, survival who underwent bone marrow transplant (BMT) (46.9%; P = 0.00021). JMML (CMML/JCML) not receiving BMT, time death shortest best JCML (absence constitutional karyotypic derangement, thrombocytopenia elevated Hb F). We conclude that abnormalities survive longer, Mo7 disorders are clinically morphologically heterogeneous should be grouped into single entity CMML may biological differences. Finally, BMT remains choice for primary MDS, intensive chemotherapy is no than supportive measures. Am. J. Hematol. 63:212–222, 2000. © 2000 Wiley-Liss, Inc.