Modeling Marrow Failure and MDS for Novel Therapeutics
Technical Report,15 Feb 2016,14 Feb 2017
Children's Hospital Corporation Boston United States
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Clonal evolution is a potentially life threatening long-term complication of inherited and acquired bone marrow failure BMF disorders. Cytogenetic clonal abnormalities develop in approximately 10 percent of patients with acquired aplastic anemia. The risk of early progression to myelodysplastic syndrome MDSand leukemia is also markedly elevated in patients with inherited marrow failure syndromes compared to age-matched controls. Prognosis of different clonal abnormalities is variable. Somatically acquired clonal deletions of one copy of chromosome 7 monosomy 7 or part of the long arm of chromosome 7del7q are generally associated with a poor prognosis. Deletions involving chromosome 7 are frequently associated with therapy-related MDS, hypocellular MDS, MDS arising in children, and in MDS arising inpatients with marrow failure.
- Medicine and Medical Research