Korean J Hematol 1997; 32(1):
Published online March 31, 1997
© The Korean Society of Hematology
김성철, 이석, 정소영, 유내춘, 민유홍, 한지숙, 고윤웅
연세대학교 의과대학 내과학교실
Background: There has been much evidence that immune-mediated stem cell injury may have a significant role in the pathogenesis of aplastic anemia, and as a result
immunosuppressive therapy has become known as an effective treatment for patients with aplastic anemia. There have been no reports regarding the long-term follow-up of
immunosuppressive therapy of patients with aplastic anemia in Korea. Therefore, we evaluated the response to immunosuppressive therapy for 47 patients with aplastic
anemia, investigating the long-term survival, relapse rate and secondary clonal hematologic diseases.
Methods: Antithymocyte globulin(ATG) or antilymphocyte globulin(ALG) was given with cyclosporin A(CsA) to 39 patients(ATG+CsA 27, ALG+CsA 12), and to 8 patients,
ATG or ALG alone was given(ATG 6, ALG 2). ATG was administered for 5(or 8) days, and ALG was administered for 5 days. CsA was orally begun with ATG or ALG for a median total of 4.5(3∼14) months.
Results:
1) Response: Among 47 patients, 30(63.8%) responded; 11 showed complete response(CR) and 19 showed partial response(PR).
2) Factors affecting response: Responses were equally distributed when patients were stratified for age, neutrophil counts and drug regimens.
3) Survival: Median duration of follow-up was 36(6∼84) months. Actuarial survival at 1 year was 100% in responders and 7.6% in nonresponders, and the 7-year actuarial
survival rate was 94% and 76%, respectively(P value = 0.13).
4) Relapse: Relapse occurred in 4 of 30 responding patients. Relapse in patients with CR was not observed during follow-up. The risk of relapse was 12% at 2 years and
22% at 6 years.
5) Treatment outcome according to disease duration: There was no significant difference in response rate between patients treated within 4 months and beyond 4 months after diagnosis. But the latter group showed a significantly higher relapse rate than the former(4% vs 60%; P value = 0.01).
6) Side effects and complications: There were no serious side effects requiring discontinuation of immunosuppressive therapy. Evolution to secondary clonal hematologic
diseases was not observed during follow-up.
Conclusion: Our results of immunosuppressive therapy show the excellent long-term outcome. A prospective study is needed for the establishment of the adequate treatment
duration of CsA and the follow-up period for the evaluation of the response of treatment.
Keywords Aplastic anemia; Immunosuppressive therapy;
Korean J Hematol 1997; 32(1): 67-78
Published online March 31, 1997
Copyright © The Korean Society of Hematology.
김성철, 이석, 정소영, 유내춘, 민유홍, 한지숙, 고윤웅
연세대학교 의과대학 내과학교실
Seong Cheol Kim, Seok Lee, So Young Chong, Nae Choon Yoo, Yoo Hong Min, Jee Sook Hahn, Yun Woong Ko
Department of Internal Medicine, Yonsei University College of Medicine, Seoul, Korea
Background: There has been much evidence that immune-mediated stem cell injury may have a significant role in the pathogenesis of aplastic anemia, and as a result
immunosuppressive therapy has become known as an effective treatment for patients with aplastic anemia. There have been no reports regarding the long-term follow-up of
immunosuppressive therapy of patients with aplastic anemia in Korea. Therefore, we evaluated the response to immunosuppressive therapy for 47 patients with aplastic
anemia, investigating the long-term survival, relapse rate and secondary clonal hematologic diseases.
Methods: Antithymocyte globulin(ATG) or antilymphocyte globulin(ALG) was given with cyclosporin A(CsA) to 39 patients(ATG+CsA 27, ALG+CsA 12), and to 8 patients,
ATG or ALG alone was given(ATG 6, ALG 2). ATG was administered for 5(or 8) days, and ALG was administered for 5 days. CsA was orally begun with ATG or ALG for a median total of 4.5(3∼14) months.
Results:
1) Response: Among 47 patients, 30(63.8%) responded; 11 showed complete response(CR) and 19 showed partial response(PR).
2) Factors affecting response: Responses were equally distributed when patients were stratified for age, neutrophil counts and drug regimens.
3) Survival: Median duration of follow-up was 36(6∼84) months. Actuarial survival at 1 year was 100% in responders and 7.6% in nonresponders, and the 7-year actuarial
survival rate was 94% and 76%, respectively(P value = 0.13).
4) Relapse: Relapse occurred in 4 of 30 responding patients. Relapse in patients with CR was not observed during follow-up. The risk of relapse was 12% at 2 years and
22% at 6 years.
5) Treatment outcome according to disease duration: There was no significant difference in response rate between patients treated within 4 months and beyond 4 months after diagnosis. But the latter group showed a significantly higher relapse rate than the former(4% vs 60%; P value = 0.01).
6) Side effects and complications: There were no serious side effects requiring discontinuation of immunosuppressive therapy. Evolution to secondary clonal hematologic
diseases was not observed during follow-up.
Conclusion: Our results of immunosuppressive therapy show the excellent long-term outcome. A prospective study is needed for the establishment of the adequate treatment
duration of CsA and the follow-up period for the evaluation of the response of treatment.
Keywords: Aplastic anemia, Immunosuppressive therapy,
Daehun Kwag, Sung‑Soo Park, Sung‑Eun Lee, Hee‑Je Kim and Jong Wook Lee
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