title: Rabbit-antithymocyte globulin combined with cyclosporin A as a first-line therapy: improved, effective, and safe for children with acquired severe aplastic anemia.
abstract: OBJECTIVE
  Acquired aplastic anemia is an organ-specific auto-immune disease characterized by pancytopenia and hypoplastic bone marrow. Immunosuppression with anti-thymocyte globulin (ATG) and cyclosporine A (CsA) is an effective and safe therapy for patients without undergoing hematopoietic stem cell transplantation. The aim of the current study was to investigate the effect of rabbit-ATG (r-ATG) combined with CsA as an intensive immunosuppressive therapy (IST) for acquired severe aplastic anemia (SAA) in children.
  
  
  METHODS
  From January 2003 to November 2008, 46 children (30 boys and 16 girls), with a median age of 7 years (between 2 and 15 years) were diagnosed with acquired SAA. They received an IST of r-ATG combined with CsA. The average time was 3.4 months (ranging from 1 to 13 months). The effective rates 3, 6, 9, and 12 months after treatment were 30.4, 65.2, 78.8, and 84.8%, respectively. After 2 years of follow-up, the response rate was 84.8% (39/46). No response was found in five cases and relapse was found in two.
  
  
  RESULTS
  Among the five cases without response, two received unrelated hematopoietic stem cell transplantation and are already disease-free and two died from infection caused by long-term dependence on infusion. No myelodysplastic syndrome or acute myeloid leukemia was found among the patients.
  
  
  CONCLUSIONS
  We propose that r-ATG combined with CsA as an intensive IST is effective and safe in treating acquired SAA in children.
authors.affiliation: Department of Pediatrics, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, No. 107, Yanjiang West Road, Guangzhou 510120, China. chunchencn@163.com
authors.forename: Chun
authors.initials: C
authors.lastname: Chen
authors.forename: Hong-Man
authors.initials: HM
authors.lastname: Xue
authors.forename: Hong-Gui
authors.initials: HG
authors.lastname: Xu
authors.forename: Yang
authors.initials: Y
authors.lastname: Li
authors.forename: Ke
authors.initials: K
authors.lastname: Huang
authors.forename: Dun-Hua
authors.initials: DH
authors.lastname: Zhou
authors.forename: Hai-Xia
authors.initials: HX
authors.lastname: Guo
authors.forename: Jian-Pei
authors.initials: JP
authors.lastname: Fang
authors.forename: Shao-Liang
authors.initials: SL
authors.lastname: Huang
mesh_terms: D000293:Adolescent; D000741:Anemia, Aplastic / Q000188:drug therapy* / Q000276:immunology; D000818:Animals; D000961:Antilymphocyte Serum / Q000008:administration & dosage* / Q000009:adverse effects; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000008:administration & dosage* / Q000009:adverse effects; D005260:Female; D006801:Humans; D007165:Immunosuppression Therapy; D007166:Immunosuppressive Agents / Q000008:administration & dosage*; D008297:Male; D011817:Rabbits; D016896:Treatment Outcome
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine