title: Treatment of severe aplastic anemia with antithymocyte globulin and cyclosporin A with or without G-CSF in adults: a multicenter randomized study in Japan.
abstract: We report the results of a randomized study to elucidate whether addition of granulocyte colony-stimulating factor (G-CSF) to immunosuppressive therapy is valuable for the treatment of severe aplastic anemia (SAA) in adults. A total of 101 previously untreated patients (median age, 54 years; range, 19 to 75 years) were randomized to receive antithymocyte globulin (ATG) and cyclosporin A (CyA) (G-CSF- group) or ATG, CyA, and G-CSF (G-CSF+ group). In the G-CSF+ group, the hematologic response rate at 6 months was higher (77% vs 57%; P = .03) than in the G-CSF- group. No differences were observed between the groups in terms of the incidence of infections and febrile episodes. There were no differences between the G-CSF- group and the G-CSF+ group in terms of survival (88% vs 94% at 4 years), and the development of myelodysplastic syndrome (MDS)/acute leukemia (AL) (1 patient vs 2 patients). However, the relapse rate was lower in the G-CSF+ group compared with the G-CSF- group (42% vs 15% at 4 years; P = .01). Further follow-up is required to elucidate the role of G-CSF in immunosuppressive therapy for adult SAA.
authors.affiliation: Department of Hematology, Tokyo Women's Medical University, Tokyo. terumura@dh.twmu.ac.jp
authors.forename: Masanao
authors.initials: M
authors.lastname: Teramura
authors.forename: Akiro
authors.initials: A
authors.lastname: Kimura
authors.forename: Satsuki
authors.initials: S
authors.lastname: Iwase
authors.forename: Yuji
authors.initials: Y
authors.lastname: Yonemura
authors.forename: Shinji
authors.initials: S
authors.lastname: Nakao
authors.forename: Akio
authors.initials: A
authors.lastname: Urabe
authors.forename: Mitsuhiro
authors.initials: M
authors.lastname: Omine
authors.forename: Hideaki
authors.initials: H
authors.lastname: Mizoguchi
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000741:Anemia, Aplastic / Q000188:drug therapy* / Q000453:epidemiology; D000961:Antilymphocyte Serum / Q000627:therapeutic use*; D016572:Cyclosporine / Q000627:therapeutic use*; D004359:Drug Therapy, Combination; D005260:Female; D016179:Granulocyte Colony-Stimulating Factor / Q000627:therapeutic use*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007564:Japan / Q000453:epidemiology; D008297:Male; D008875:Middle Aged; D015996:Survival Rate; D016896:Treatment Outcome
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016179:Granulocyte Colony-Stimulating Factor; D016572:Cyclosporine