韩国儿童再生障碍性贫血15年的流行病学及临床长期转归：韩国儿科血液肿瘤学会（KSPHO）的回顾性研究

Epidemiology and clinical long-term outcome of childhood aplastic anemia in Korea for 15 years: retrospective study of the Korean Society of Pediatric Hematology Oncology (KSPHO).

作者信息

Jeong Dae Chul, Chung Nack Gyun, Kang Hyoung Jin, Koo Hong Hoe, Kook Hoon, Kim Soon Ki, Kim Sun Young, Kim Heung Sik, Kim Hwang Min, Park Kyung Duk, Park Sang Kyu, Park Jae Sun, Park Jun Eun, Park Hyeon Jin, Park Young Shil, Seo Jong Jin, Sung Ki Woong, Shin Hee Young, Ahn Hyo Seop, Ryu Kun Hee, Ryu Kyung Ha, Yoo Eun Sun, Lyu Chuhl Joo, Lee Kun Soo, Lee Kwang Chul, Lee Soon Yong, Lee Young Ho, Lim Young Tak, Lim Yeon Jung, Jung Hye Lim, Cho Bin, Choi Yong Mook, Hah Jeong Ok, Hwang Tai Ju, Kim Hack Ki

机构信息

Department of Pediatrics, College of Medicine, The Catholic University of Korea, Korea.

出版信息

J Pediatr Hematol Oncol. 2011 Apr;33(3):172-8. doi: 10.1097/MPH.0b013e31820826a8.

DOI:10.1097/MPH.0b013e31820826a8

PMID:21325968

Abstract

PURPOSE

Aplastic anemia (AA) is a rare hematologic disease characterized by pancytopenia and hypocellular marrow. The Korean Society of Pediatric Hematology Oncology investigated retrospectively the incidence, survival, and transfusion independency according to treatment strategies in AA.

METHODS

All the questionnaires were sent to members for medical records. We collected and analyzed 702 available data.

RESULTS

The male and female ratio was 1.2, and the median age at diagnosis was 9.3 years. The annual incidence of Korean children with AA was 5.16 per million per year. Constitutional anemia was diagnosed in 44 children. In acquired AA, causes were identified in 39 children. Severe AA (SAA) at initial diagnosis was more common than nonsevere AA. The overall survival was 47.8% with supportive care, 68.1% with immunosuppressive therapy (IST), and 81.8% with hematopoietic stem cell transplantation. In IST, response rate was 65.7%, and relapse rate after response was 54.4% within a median of 23.0 months. The factors with overall survival were severity of disease in supportive care, severity and response in IST, donor type, graft failure, and posttransplant events in hematopoietic stem cell transplantation.

CONCLUSIONS

Long-term outcome in AA was dependent on treatment strategies. These Korean results may help research and prospective international clinical trials for childhood AA.

摘要

目的

再生障碍性贫血（AA）是一种罕见的血液系统疾病，其特征为全血细胞减少和骨髓细胞减少。韩国儿科学血液肿瘤学会根据AA的治疗策略，对其发病率、生存率和输血独立性进行了回顾性研究。

方法

向会员发送所有问卷以获取病历。我们收集并分析了702份可用数据。

结果

男女比例为1.2，诊断时的中位年龄为9.3岁。韩国儿童AA的年发病率为每年百万分之5.16。44名儿童被诊断为先天性贫血。在获得性AA中，39名儿童明确了病因。初诊时重度AA（SAA）比非重度AA更常见。支持治疗的总生存率为47.8%，免疫抑制治疗（IST）为68.1%，造血干细胞移植为81.8%。在IST中，缓解率为65.7%，缓解后复发率在中位23.0个月内为54.4%。支持治疗中总生存的因素包括疾病严重程度、IST中的严重程度和缓解情况、供体类型、移植物失败以及造血干细胞移植后的移植后事件。