Rabbit-antithymocyte globulin combined with cyclosporin A as a first-line therapy: improved, effective, and safe for children with acquired severe aplastic anemia. | Aplastic Anemia and MDS International Foundation

Rabbit-antithymocyte globulin combined with cyclosporin A as a first-line therapy: improved, effective, and safe for children with acquired severe aplastic anemia.

Journal Title: 
J Cancer Res Clin Oncol
Author(s): 
Chen C, Xue HM, Xu HG, Li Y, Huang K, Zhou DH, Guo HX, Fang JP, Huang SL.
Primary Author: 
Chen C
Original Publication Date: 
Saturday, March 10, 2012

PURPOSE:

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.

Bone Marrow Diseases: