title: Rabbit antithymocyte globulin as first-line therapy for severe aplastic anemia.
abstract: Rabbit antithymocyte globulin (rATG) was proven effective as salvage therapy for refractory aplastic anemia (AA), or for relapse after initial therapy with horse ATG (hATG). Several clinical trials were performed to assess the efficiency of rATG as a first-line therapy for AA patients; however, their results were variable. The aim of the present study was to assess hematologic response and survival in severe AA (SAA) and very severe AA (VSAA) patients treated with rATG and cyclosporin A (CsA) in our center. The factors involved in these outcomes were also explored. A total of 292 patients with newly diagnosed, acquired SAA or VSAA received a combination of rATG and CsA as first-line therapy, and the results were retrospectively assessed. The median age was 18 years (range = 2-73 years). The early death rate was 5.5%, and the total response rates were 49.0% (143 responders), 60.3% (176 responders), 65.8% (192 responders), and 68.5% (200 responders) at 3, 6, 9, and 12 months, respectively, after immunosuppressive therapy. In multivariate analysis, initial response to granulocyte colony-stimulating factor (G-CSF) was the predictive factor for response to therapy at 12 months. Median follow-up of surviving patients was 34 months (range = 0-117 months). Five-year overall survival was 83.2%, and the 5-year, event-free survival was 67.2%. Independent prognostic factors for overall survival were neutrophil count and achievement of any response following rATG therapy. Our results indicate that rATG/CsA is a safe and effective first-line treatment for SAA/VSAA.
authors.affiliation: Department of Anemia Therapeutic Centre, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Science & Peking Union Medical College (CAMS & PUMC), Tianjin, China.
authors.forename: Li
authors.initials: L
authors.lastname: Zhang
authors.affiliation: Department of Anemia Therapeutic Centre, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Science & Peking Union Medical College (CAMS & PUMC), Tianjin, China.
authors.forename: Liping
authors.initials: L
authors.lastname: Jing
authors.affiliation: Department of Anemia Therapeutic Centre, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Science & Peking Union Medical College (CAMS & PUMC), Tianjin, China.
authors.forename: Kang
authors.initials: K
authors.lastname: Zhou
authors.affiliation: Department of Pathology, Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC, Tianjin, China.
authors.forename: Huijun
authors.initials: H
authors.lastname: Wang
authors.affiliation: Department of Pathology, Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC, Tianjin, China.
authors.forename: Guangxin
authors.initials: G
authors.lastname: Peng
authors.affiliation: Department of Anemia Therapeutic Centre, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Science & Peking Union Medical College (CAMS & PUMC), Tianjin, China.
authors.forename: Yang
authors.initials: Y
authors.lastname: Li
authors.affiliation: Department of Anemia Therapeutic Centre, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Science & Peking Union Medical College (CAMS & PUMC), Tianjin, China.
authors.forename: Yuan
authors.initials: Y
authors.lastname: Li
authors.affiliation: Department of Anemia Therapeutic Centre, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Science & Peking Union Medical College (CAMS & PUMC), Tianjin, China.
authors.forename: Jianping
authors.initials: J
authors.lastname: Li
authors.affiliation: Department of Anemia Therapeutic Centre, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Science & Peking Union Medical College (CAMS & PUMC), Tianjin, China.
authors.forename: Lei
authors.initials: L
authors.lastname: Ye
authors.affiliation: Department of Anemia Therapeutic Centre, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Science & Peking Union Medical College (CAMS & PUMC), Tianjin, China.
authors.forename: Lihui
authors.initials: L
authors.lastname: Shi
authors.affiliation: Department of Anemia Therapeutic Centre, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Science & Peking Union Medical College (CAMS & PUMC), Tianjin, China.
authors.forename: Huihui
authors.initials: H
authors.lastname: Fan
authors.affiliation: Department of Anemia Therapeutic Centre, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Science & Peking Union Medical College (CAMS & PUMC), Tianjin, China.
authors.forename: Xin
authors.initials: X
authors.lastname: Zhao
authors.affiliation: Department of Leukemia Therapeutic Centre, State Key Laboratory of Experimental Hematology, Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC, Tianjin, China.
authors.forename: Jianxiang
authors.initials: J
authors.lastname: Wang
authors.affiliation: Department of Anemia Therapeutic Centre, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Science & Peking Union Medical College (CAMS & PUMC), Tianjin, China. Electronic address: ZhFK@Hotmail.com.
authors.forename: Fengkui
authors.initials: F
authors.lastname: Zhang
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000741:Anemia, Aplastic / Q000188:drug therapy*; D000818:Animals; D000961:Antilymphocyte Serum / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D005260:Female; D016179:Granulocyte Colony-Stimulating Factor / Q000008:administration & dosage; D006801:Humans; D008297:Male; D008875:Middle Aged; D011817:Rabbits; D016879:Salvage Therapy; D012720:Severity of Illness Index; D016019:Survival Analysis; D055815:Young Adult
chemical_list: D000961:Antilymphocyte Serum; D016179:Granulocyte Colony-Stimulating Factor