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

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.