title: [A long-term follow up study on 345 severe aplastic anemia patients treated with antithymocyte globulin/lymphoglobulin].
abstract: OBJECTIVE
  To assess the short term curative efficacy and long-term survival outcomes of severe aplastic anemia patients following antithymocyte globulin/lymphoglobulin (ATG/ALG) with or without cyclosporine (CsA).
  
  
  METHODS
  A total of 345 cases hospitalized in our hospital between December 1982 and June 2011 were enrolled into this study. We assessed the response rates 3 and 6 months after ATG/ALG, and estimated the overall survival (OS) by Kaplan-Meier method for this cohort of patients.
  
  
  RESULTS
  The cohort of 345 patients was routinely followed-up with a median follow-up of 44.0 (range, 0.5 - 244.0) months. The response rates at 3 and 6 months were 29.9% and 45.4%, respectively. The differences in response rates at both 3 (39.2% vs 19.6%, P < 0.01) and 6 months (55.6% vs 34.0%, P < 0.01) between 184 non-severe aplastic anemia (mSAA) and 161 very severe aplastic anemia (VSAA) were statistically significant. The response rates among the different ATG preparations were comparative; but 3-(10.6%) and 6-month (25.5%) responses produced by rATG-Fresenius were significantly inferior to those by rATG-Sangstat (36.6% and 56.6%, respectively) (all P < 0.01). The 5-year OS was 61.7% (95%CI 55.4% - 68.0%) for the entire cohort of patients, and 5-year OS for mSAA patients \[71.0% (95%CI 62.9% - 79.1%)\] was superior to that of VSAA patients \[50.4% (95%CI 40.1% - 60.7%), P < 0.01\]; but for the patients treated from 2007, the difference of OS in the last 5 years between VSAA and mSAA was not significant \[ 73.7% (95%CI 52.2% - 95.2%) vs 89.7% (95%CI 79.5% - 99.9%); P = 0.24\]. Our study also confirmed the superiority of ATG/ALG + CsA regimen \[64.8% (95%CI 57.9% - 71.7%)\] over ATG/ALG alone \[32.6% (95%CI 15.7% - 49.5%)\] with regard to 5-year OS (P < 0.01); but the addition of recombinant human granulocyte colony-stimulating factor (rhG-CSF) to ATG/ALG had no benefit in terms of OS. rATG-S produced significantly better 5-year OS \[66.1% (95%CI 55.8% - 76.4%)\] than rATG-F \[46.6% (95%CI 35.9% - 57.3%); P < 0.01\].
  
  
  CONCLUSIONS
  (1) The outcome of mSAA was superior to that of VSAA, but the latter was markedly improved in the last 5 years; (2) rATG-F was inferior to rATG-S with regard to 5-year OS; (3) Immunosuppressive treatment with ATG/ALG plus CsA was more effective than ATG/ALG alone; (4) The addition of rhG-CSF to ATG/ALG had no benefit in terms of OS.
authors.affiliation: Institute of Hematology & Blood Diseases Hospital, CAMS & PUMC, Tianjin, China.
authors.forename: Ying-qi
authors.initials: YQ
authors.lastname: Shao
authors.forename: Xing-xin
authors.initials: XX
authors.lastname: Li
authors.forename: Mei-li
authors.initials: ML
authors.lastname: Ge
authors.forename: Jun
authors.initials: J
authors.lastname: Shi
authors.forename: Jing
authors.initials: J
authors.lastname: Zhang
authors.forename: Jin-bo
authors.initials: JB
authors.lastname: Huang
authors.forename: Zhen-dong
authors.initials: ZD
authors.lastname: Huang
authors.forename: Neng
authors.initials: N
authors.lastname: Nie
authors.forename: Yi-zhou
authors.initials: YZ
authors.lastname: Zheng
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000741:Anemia, Aplastic / Q000628:therapy*; D000961:Antilymphocyte Serum / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007136:Immunoglobulins / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D011379:Prognosis; D012189:Retrospective Studies; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D000961:Antilymphocyte Serum; D007136:Immunoglobulins