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

Journal of the Korean Pediatric Society 1998;41(6):775-784.
Published online June 15, 1998.
Immunomodulation Therapy in Aplastic Anemia : Relapse Rate, Complications and Long-term Survival During Follow-up for More than 1 Year
Jun Ah Lee, Hyoung Jin Kang, Hyo Jeong Han, Hyoung Soo Choi, Eun Sun Yoo, Hee Young Shin, Joong Gon Kim, Hyo Seop Ahn
Department of Pediatrics, Seoul National University College of Medicine Seoul, Korea
재생불량성 빈혈에서의 면역조절 요법 - 1년 이상 추적 관찰시의 재발률, 합병증, 장기 생존율 -
이준아, 강형진, 한효정, 최형수, 유은선, 신희영, 김중곤, 안효섭
서울대학교 의과대학 소아과학교실
Correspondence: 
Hyo Seop Ahn, Email: 1
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.
Key Words: Aplastic anemia, Immunomodulation therapy, Antilymphocyte globulin, Antithymocyte globulin, Cyclosporine A


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