Antileukemic activity of the azacitidine in a xenograft model of juvenile myelomonocytic leukemia
CF Krombholz, S Fluhr, CM Niemeyer, C Flotho, M Erlacher · Klinische Pädiatrie · 2016 · Heft 05
Bibliografische Angaben
Zusammenfassung
Introduction: Juvenile myelomonocytic leukemia (JMML) is an aggressive myeloproliferative disorder of early childhood. Patients present with hepatosplenomegaly, lymphadenopathy and skin rash, and peripheral blood analysis reveals monocytosis and the presence of myeloid precursors. Clinical signs are caused by organ infiltration of proliferating monocytes and granulocytes. JMML cells are characterized by a deregulated activation of the RAS signaling pathway as a result of mutations in KRAS, NRAS, NF1, PTPN11 (encoding SHP2) or CBL. When left untreated, most patients die from respiratory failure due to leukemic lu…