Molecular and cellular biological study on the mechanism of spontaneous remission of transient myeloproliferative disorder in children with Down syndrome

唐氏综合征新生儿一过性骨髓增生性疾病（TMD）是一种特殊的白血病，发生于胎儿期，出生后自发消退。我们研究了造血微环境在TMD自发缓解中的作用。在生长因子的存在下，TMD胚细胞分化成嗜碱性粒细胞/肥大细胞和巨核细胞谱系和异常表达的GATA 1蛋白由于GATA 1突变被证明是在分化过程中下调。TMD母细胞的生长由胎肝基质细胞维持。提示TMD母细胞的分化潜能和胎儿造血微环境可能参与TMD的自发缓解。

Transient myeloproliferative disorder (TMD) in neonates with Down syndrome is a special leukemia that arises in fetal stage and spontaneously regresses after birth. We studied the role of hematopoietic microenvironment in spontaneous remission of TMD. In the presence of growth factors, TMD blasts differentiated into basophil/mast cell and megakaryocyte lineages and aberrant expression of GATA1 protein due to GATA1 mutations was shown to be down-regulated during differentiation. The growth of TMD blasts was maintained by stromal cells of the fetal liver. These data indicate that differentiation potential of TMD blasts and fetal hematopoietic microenvironment could be involved in spontaneous remission of TMD.