title: Management of hypereosinophilic syndrome: a prospective study in the era of molecular genetics.
abstract: Hypereosinophilic syndrome (HES) is a heterogeneous group of disorders characterized by unexplained persistent primary eosinophilia causing end-organ damage. We conducted a prospective cohort study of patients fulfilling the diagnostic criteria for HES. Of 20 patients considered eligible for the study, 2 were found to have clonal myeloid disorders, limiting the diagnosis of "true" HES to 18 patients. No patient carried the FIP1L1-PDGFRA fusion gene or other imatinib-responsive translocations. A clonal interleukin-5-producing T-cell population was not detected in any patient. Common manifestations at presentation were pulmonary, cutaneous, and neurologic involvement; serositis; and gastrointestinal involvement. Only 3 patients developed cardiac involvement. Fifteen of the HES patients were administered first-line combined treatment with steroids and hydroxyurea. Nine patients achieved complete response, while 6 attained only partial response. Imatinib was administered to 3 HES patients who had been pretreated with steroids, resulting in complete hematologic and clinical response in 2 patients and no response at all in 1. Further treatment of the latter patient with steroids and hydroxyurea also proved ineffective. We conclude that the therapeutic approach should be individualized according to molecular findings. We consider the coadministration of corticosteroids and hydroxyurea to be an effective combination for the treatment of FIP1L1-PDGFRA-negative HES.
authors.affiliation: From Department of Pathophysiology (IJD, SG, MV, HMM), Medical School, National University of Athens; Hematology Research Laboratory (CZ, KZ), Biomedical Research Foundation, Academy of Athens, Athens, Greece.
authors.forename: Issa J
authors.initials: IJ
authors.lastname: Dahabreh
authors.forename: Stavroula
authors.initials: S
authors.lastname: Giannouli
authors.forename: Christine
authors.initials: C
authors.lastname: Zoi
authors.forename: Katerina
authors.initials: K
authors.lastname: Zoi
authors.forename: Michael
authors.initials: M
authors.lastname: Voulgarelis
authors.forename: Haralampos M
authors.initials: HM
authors.lastname: Moutsopoulos
mesh_terms: D000328:Adult; D000368:Aged; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use; D001549:Benzamides; D020732:Cytogenetic Analysis; D005260:Female; D006801:Humans; D006918:Hydroxyurea / Q000008:administration & dosage; D017681:Hypereosinophilic Syndrome / Q000175:diagnosis* / Q000188:drug therapy* / Q000235:genetics; D000068877:Imatinib Mesylate; D008297:Male; D008875:Middle Aged; D015514:Oncogene Proteins, Fusion / Q000235:genetics*; D018579:Patient Selection; D010879:Piperazines / Q000627:therapeutic use; D011241:Prednisone / Q000008:administration & dosage; D011446:Prospective Studies; D011743:Pyrimidines / Q000627:therapeutic use; D020796:Receptor, Platelet-Derived Growth Factor alpha / Q000235:genetics*; D014178:Translocation, Genetic / Q000235:genetics; D039221:mRNA Cleavage and Polyadenylation Factors / Q000235:genetics*
chemical_list: D000970:Antineoplastic Agents; D001549:Benzamides; D015514:Oncogene Proteins, Fusion; D010879:Piperazines; D011743:Pyrimidines; D039221:mRNA Cleavage and Polyadenylation Factors; D000068877:Imatinib Mesylate; C479819:FIP1L1-PDGFRA fusion protein, human; D020796:Receptor, Platelet-Derived Growth Factor alpha; D011241:Prednisone; D006918:Hydroxyurea