title: Imatinib has limited therapeutic activity for hypereosinophilic syndrome patients with unknown or negative PDGFRalpha mutation status.
abstract: Hypereosinophilic syndrome (HES) is characterized by sustained non-clonal blood and tissue eosinophilia, leading to end-organ damage. With a molecular/cytogenetic clonality marker, the disease is classified as chronic eosinophilic leukemia (CEL). Efficacy of imatinib mesylate is well established in CEL with FIP1L1-platelet-derived growth factor-alpha (PDGFRalpha) rearrangement. We treated with imatinib 18 HES patients (11 PDGFRalpha-negative and 7 PDGFRalpha-status unknown). One patient with unknown PDGFRalpha status achieved complete hematologic response, and two (one PDGFRalpha negative and one status unknown) achieved partial hematologic response. Our results confirm low response rate to imatinib in HES patients with unknown or negative PDGFRalpha status, and underscore the need for new therapeutic options for this disorder.
authors.affiliation: Department of Leukemia, University of Texas, M.D. Anderson Cancer Center, Houston, TX 77030, USA.
authors.forename: Nitin
authors.initials: N
authors.lastname: Jain
authors.forename: Jorge
authors.initials: J
authors.lastname: Cortes
authors.forename: Alfonso
authors.initials: A
authors.lastname: Quintás-Cardama
authors.forename: Taghi
authors.initials: T
authors.lastname: Manshouri
authors.forename: Raja
authors.initials: R
authors.lastname: Luthra
authors.forename: Guillermo
authors.initials: G
authors.lastname: Garcia-Manero
authors.forename: Hagop
authors.initials: H
authors.lastname: Kantarjian
authors.forename: Srdan
authors.initials: S
authors.lastname: Verstovsek
mesh_terms: D000328:Adult; D000368:Aged; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D001549:Benzamides; D005260:Female; D006801:Humans; D017681:Hypereosinophilic Syndrome / Q000188:drug therapy*; D000068877:Imatinib Mesylate; D008297:Male; D008875:Middle Aged; D009154:Mutation; D010879:Piperazines / Q000627:therapeutic use*; D011743:Pyrimidines / Q000627:therapeutic use*; D020796:Receptor, Platelet-Derived Growth Factor alpha / Q000235:genetics*
chemical_list: D000970:Antineoplastic Agents; D001549:Benzamides; D010879:Piperazines; D011743:Pyrimidines; D000068877:Imatinib Mesylate; D020796:Receptor, Platelet-Derived Growth Factor alpha