title: Outcomes of optimized over standard protocol of rabbit antithymocyte globulin for severe aplastic anemia: a single-center experience.
abstract: BACKGROUND
  Previous reports showed that outcome of rabbit antithymocyte globulin (rATG) was not satisfactory as the first-line therapy for severe aplastic anemia (SAA). We explored a modifying schedule of administration of rATG.
  
  
  METHODS
  Outcomes of a cohort of 175 SAA patients, including 51 patients administered with standard protocol (3.55 mg/kg/d for 5 days) and 124 cases with optimized protocol (1.97 mg/kg/d for 9 days) of rATG plus cyclosporine (CSA), were analyzed retrospectively.
  
  
  RESULTS
  Of all 175 patients, response rates at 3 and 6 months were 36.6% and 56.0%, respectively. 51 cases received standard protocol had poor responses at 3 (25.5%) and 6 months (41.2%). However, 124 patients received optimized protocol had better responses at 3 (41.1%, P = 0.14) and 6 (62.1%, P = 0.01). Higher incidences of infection (57.1% versus 37.9%, P = 0.02) and early mortality (17.9% versus 0.8%, P<0.001) occurred in patients received standard protocol compared with optimized protocol. The 5-year overall survival in favor of the optimized over standard rATG protocol (76.0% versus. 50.3%, P<0.001) was observed. By multivariate analysis, optimized protocol (RR = 2.21, P = 0.04), response at 3 months (RR = 10.31, P = 0.03) and shorter interval (<23 days) between diagnosis and initial dose of rATG (RR = 5.35, P = 0.002) were independent favorable predictors of overall survival.
  
  
  CONCLUSIONS
  Optimized instead of standard rATG protocol in combination with CSA remained efficacious as a first-line immunosuppressive regimen for SAA.
authors.affiliation: Severe Aplastic Anemia Studying Program, State Key Laboratory of Experimental Hematology, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Science & Peking Union Medical College, Tianjin, PR China.
authors.forename: Xingxin
authors.initials: X
authors.lastname: Li
authors.forename: Jun
authors.initials: J
authors.lastname: Shi
authors.forename: Meili
authors.initials: M
authors.lastname: Ge
authors.forename: Yingqi
authors.initials: Y
authors.lastname: Shao
authors.forename: Jinbo
authors.initials: J
authors.lastname: Huang
authors.forename: Zhendong
authors.initials: Z
authors.lastname: Huang
authors.forename: Jing
authors.initials: J
authors.lastname: Zhang
authors.forename: Neng
authors.initials: N
authors.lastname: Nie
authors.forename: Yizhou
authors.initials: Y
authors.lastname: Zheng
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000175:diagnosis / Q000188:drug therapy* / Q000401:mortality*; D000818:Animals; D000961:Antilymphocyte Serum / Q000008:administration & dosage*; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000008:administration & dosage; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage*; D008297:Male; D008875:Middle Aged; D011817:Rabbits; D012189:Retrospective Studies; D015996:Survival Rate
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine
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