title: Predicting response to immunosuppressive therapy in childhood aplastic anemia.
abstract: In aplastic anemia, predictive markers of response to immunosuppressive therapy have not been well defined. We retrospectively evaluated whether clinical and laboratory findings before treatment could predict response in a pediatric cohort from the multicenter AA-97 study in Japan. Between 1997 and 2006, 312 newly diagnosed children were enrolled and treated with a combination of antithymocyte globulin and cyclosporine. In multivariate analyses, lower white blood cell count was the most significant predictive marker of better response; patients with white blood cell count less than 2.0×10(9)/L showed a higher response rate than those with white blood cell count of 2.0×10(9)/L or more (P=0.0003), followed by shorter interval between diagnosis and therapy (P=0.01), and male sex (P=0.03). In conclusion, pre-treatment clinical and laboratory findings influence response to therapy. The finding that response rate worsens with increasing interval between diagnosis and treatment highlights the importance of prompt immunosuppressive therapy for patients with aplastic anemia.
authors.affiliation: Department of Hematology and Oncology, Children’s Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
authors.forename: Nao
authors.initials: N
authors.lastname: Yoshida
authors.forename: Hiroshi
authors.initials: H
authors.lastname: Yagasaki
authors.forename: Asahito
authors.initials: A
authors.lastname: Hama
authors.forename: Yoshiyuki
authors.initials: Y
authors.lastname: Takahashi
authors.forename: Yoshiyuki
authors.initials: Y
authors.lastname: Kosaka
authors.forename: Ryoji
authors.initials: R
authors.lastname: Kobayashi
authors.forename: Hiromasa
authors.initials: H
authors.lastname: Yabe
authors.forename: Takashi
authors.initials: T
authors.lastname: Kaneko
authors.forename: Masahiro
authors.initials: M
authors.lastname: Tsuchida
authors.forename: Akira
authors.initials: A
authors.lastname: Ohara
authors.forename: Tatsutoshi
authors.initials: T
authors.lastname: Nakahata
authors.forename: Seiji
authors.initials: S
authors.lastname: Kojima
mesh_terms: D000293:Adolescent; D000741:Anemia, Aplastic / Q000097:blood / Q000175:diagnosis / Q000188:drug therapy*; D000961:Antilymphocyte Serum / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000627:therapeutic use*; D004359:Drug Therapy, Combination; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D007958:Leukocyte Count; D008297:Male; D015337:Multicenter Studies as Topic; D015999:Multivariate Analysis; D011237:Predictive Value of Tests; D011379:Prognosis; D012189:Retrospective Studies; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine