Scientists at University of Oxford, Weatherall Institute of Molecular Medicine release new data on acute megakaryocytic leukemia in children

03-01-2007

Scientists discuss in "Molecular insights into Down syndrome-associated leukemia" new findings in acute megakaryocytic leukemia. According to recent research from Oxford, the United Kingdom, "Four years ago it was discovered that nearly all cases of transient myeloproliferative disorder and acute megakaryocytic leukemia in children with Down syndrome acquire mutations in the hematopoietic transcription factor gene GATA1. Studies within the past year, described within this review, have provided tremendous insights into the role of GATA1 mutations in these malignancies."

"In the past year, our understanding of the molecular and cellular consequences of GATA1 mutations has been greatly enhanced. Most importantly, we have learned that these mutations, which result in the exclusive production of the short GATA1 isoform named GATA1s, have a distinct effect on fetal liver progenitors. In addition, multiple studies have shown that GATA1s can substitute for GATA1 in many aspects of megakaryocytic maturation. Finally, an important clinical study has revealed that GATA1 mutations alone are insufficient for leukemia. Leukemia in children with Down syndrome requires at least three cooperating events--trisomy 21, a GATA1 mutation, and a third, as yet undefined, genetic alteration. Recent studies have provided tremendous insights into the GATA1 side of the story," wrote P. Vyas and colleagues, University of Oxford, Weatherall Institute of Molecular Medicine.

The researchers concluded: "Future experiments with human patient samples and mouse models will likely increase our awareness of the role of trisomy 21 in transient myeloproliferative disorder and acute megakaryocytic leukemia."

Vyas and colleagues published their study in Current Opinion In Pediatrics (Molecular insights into Down syndrome-associated leukemia. Current Opinion In Pediatrics, 2007;19(1):9-14).

For additional information, contact P. Vyas, John Radcliffe Hospital, Dept. of Haematology, MRC Molecular Haematology Unit, Weatherall Institute of Molecular Medicine, University of Oxford, Oxford, UK.

Publisher contact information for the journal Current Opinion In Pediatrics is: Lippincott Williams & Wilkins, 530 Walnut St., Philadelphia, PA 19106-3621, USA.

This article was prepared by Clinical Oncology Week editors from staff and other reports. Copyright 2007, Clinical Oncology Week via NewsRx.com.