title: 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).
abstract: OBJECTIVE
  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.
authors.affiliation: Department of Pediatrics, College of Medicine, The Catholic University of Korea, Korea.
authors.forename: Dae Chul
authors.initials: DC
authors.lastname: Jeong
authors.forename: Nack Gyun
authors.initials: NG
authors.lastname: Chung
authors.forename: Hyoung Jin
authors.initials: HJ
authors.lastname: Kang
authors.forename: Hong Hoe
authors.initials: HH
authors.lastname: Koo
authors.forename: Hoon
authors.initials: H
authors.lastname: Kook
authors.forename: Soon Ki
authors.initials: SK
authors.lastname: Kim
authors.forename: Sun Young
authors.initials: SY
authors.lastname: Kim
authors.forename: Heung Sik
authors.initials: HS
authors.lastname: Kim
authors.forename: Hwang Min
authors.initials: HM
authors.lastname: Kim
authors.forename: Kyung Duk
authors.initials: KD
authors.lastname: Park
authors.forename: Sang Kyu
authors.initials: SK
authors.lastname: Park
authors.forename: Jae Sun
authors.initials: JS
authors.lastname: Park
authors.forename: Jun Eun
authors.initials: JE
authors.lastname: Park
authors.forename: Hyeon Jin
authors.initials: HJ
authors.lastname: Park
authors.forename: Young Shil
authors.initials: YS
authors.lastname: Park
authors.forename: Jong Jin
authors.initials: JJ
authors.lastname: Seo
authors.forename: Ki Woong
authors.initials: KW
authors.lastname: Sung
authors.forename: Hee Young
authors.initials: HY
authors.lastname: Shin
authors.forename: Hyo Seop
authors.initials: HS
authors.lastname: Ahn
authors.forename: Kun Hee
authors.initials: KH
authors.lastname: Ryu
authors.forename: Kyung Ha
authors.initials: KH
authors.lastname: Ryu
authors.forename: Eun Sun
authors.initials: ES
authors.lastname: Yoo
authors.forename: Chuhl Joo
authors.initials: CJ
authors.lastname: Lyu
authors.forename: Kun Soo
authors.initials: KS
authors.lastname: Lee
authors.forename: Kwang Chul
authors.initials: KC
authors.lastname: Lee
authors.forename: Soon Yong
authors.initials: SY
authors.lastname: Lee
authors.forename: Young Ho
authors.initials: YH
authors.lastname: Lee
authors.forename: Young Tak
authors.initials: YT
authors.lastname: Lim
authors.forename: Yeon Jung
authors.initials: YJ
authors.lastname: Lim
authors.forename: Hye Lim
authors.initials: HL
authors.lastname: Jung
authors.forename: Bin
authors.initials: B
authors.lastname: Cho
authors.forename: Yong Mook
authors.initials: YM
authors.lastname: Choi
authors.forename: Jeong Ok
authors.initials: JO
authors.lastname: Hah
authors.forename: Tai Ju
authors.initials: TJ
authors.lastname: Hwang
authors.forename: Hack Ki
authors.initials: HK
authors.lastname: Kim
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000453:epidemiology* / Q000209:etiology / Q000401:mortality / Q000628:therapy; D002648:Child; D002675:Child, Preschool; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D015994:Incidence; D007223:Infant; D007723:Korea / Q000453:epidemiology; D008297:Male; D012189:Retrospective Studies; D016896:Treatment Outcome
chemical_list: D007166:Immunosuppressive Agents