Immunomodulation Therapy in Aplastic Anemia: Relapse Rate, Complications and Long-term Survival During Follow-up for More than 1 Year

Lee, Jun Ah; Kang, Hyoung Jin; Han, Hyo Jeong; Choi, Hyoung Soo; Yoo, Eun Sun; Shin, Hee Young; Kim, Joong Gon; Ahn, Hyo Seop

Abstract

PURPOSE: We evaluated the response to immunomodulation therapy, long-term survival and relapse rate of aplastic anemia during follow-up for more than 1 year.
METHODS
Twenty-eight children, with moderate to severe aplastic anemia were followed for more than one year and 7 children expired after therapy, were analyzed. Antilymphocyte globulin (ALG) or antithymocyte globulin (ATG) by itself was given to 27 patients, and cyclosporine A (CsA) combined with ALG was given to 8 patients. ALG (ATG) was administered for 8 days in 20mg/kg/day when used alone, and for 5 days in 10mg/kg/day when combined with CsA. CsA was orally administered on the 14th day till 180th day of therapy, with 5mg/kg/day for first 14 days and 3mg/kg/day thereafter.
RESULTS
Fifteen out of 35 patients (43%) showed a response. Median interval to response was 3 month (1-40 months). Response rate to ALG + CsA was 50%, compared to 41% in ALG (or ATG) alone. The interval of aplastic anemia symptoms to treatment showed a tendency to be shorter in responders (4.1 +/- 2.2 months) than in non-responders (17.5 +/- 6.4 months). Relapse occurred in 3 of 15 responders (20%). Evolution to secondary clonal hematologic disorders was not observed. Overall actuarial survival at 3 year was 78.2%.
CONCLUSION
The results of immunomodulation therapy suggest that response would be better for patients whose interval from symptoms of aplastic anemia to treatment is short. Our results also suggest that ALG+CsA would be a better treatment modality to improve response rate.