title: Mast cell leukemia with prolonged survival on PKC412/midostaurin.
abstract: Mast cell leukemia (MCL) is a rare and aggressive form of systemic mastocytosis. There are approximately 50 reported cases since 1950s. MCL is refractory to cytoreduction chemotherapy and the average survival is only six months. We report a MCL case in a 71 year-old woman with high tumor load at the initial presentation in 2005, who did not respond to either interleukin-2 or dasatinib therapy. After enrolled in a clinical trial of PKC412 (or Midostaurin) with a daily dose of 100 mg, the patient responded well to PKC412 and became transfusion independent in three months. Since then, her disease had been stably controlled. This is the first report of a high-tumor-load MCL case which achieved prolonged survival (101 months) by PKC 412. The 101-month overall survival is the longest among reported MCL cases in the English literature.
authors.affiliation: Department of Pathology, School of Medicine, University of California, San Diego; VA San Diego Healthcare System San Diego CA, USA ; Department of Pathology and Immunology, School of Medicine, Washington University St. Louis MO, USA.
authors.forename: Xiangdong
authors.initials: X
authors.lastname: Xu
authors.affiliation: Department of Pathology and Immunology, School of Medicine, Washington University St. Louis MO, USA.
authors.forename: Friederike H
authors.initials: FH
authors.lastname: Kreisel
authors.affiliation: Department of Pathology and Immunology, School of Medicine, Washington University St. Louis MO, USA.
authors.forename: John L
authors.initials: JL
authors.lastname: Frater
authors.affiliation: Department of Pathology and Immunology, School of Medicine, Washington University St. Louis MO, USA.
authors.forename: Anjum
authors.initials: A
authors.lastname: Hassan
mesh_terms: D000368:Aged; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D017322:Clinical Trials, Phase II as Topic; D005260:Female; D006801:Humans; D007946:Leukemia, Mast-Cell / Q000188:drug therapy* / Q000473:pathology*; D019311:Staurosporine / Q000031:analogs & derivatives* / Q000627:therapeutic use
chemical_list: D000970:Antineoplastic Agents; D019311:Staurosporine; C059539:midostaurin
keywords: D816V; Mast cell leukemia; Midostaurin; PKC412; aleukemic variant; c-kit; prolonged survival