title: [Clinical features of childhood refractory cytopenia].
abstract: OBJECTIVE
  To study the clinical features of patients with refractory cytopenia of childhood (RCC).
  
  
  METHODS
  The clinical data of 1 420 children (0-14 years old) with an initial diagnosis of non-severe aplastic anemia between January 1990 and June 2013 were retrospectively analyzed. Bone marrow cell morphology and histopathology were re-evaluated, and the patients were re-classified using the criteria proposed in the 2008 edition of the World Health Organization classification of RCC in hematopoietic and lymphoid tumor tissues. The clinical outcomes were followed up every 3-6 months.
  
  
  RESULTS
  Among all the 1 420 cases, 152 (10.7%) were reassessed as RCC. Patients with RCC had a lower level of hemoglobin and a higher percentage of fetal hemoglobin than those with non-severe aplastic anemia. Of the patients with RCC, 21.5% showed abnormal karyotypes at diagnosis. The median follow-up period for all patients was 36 months (ranging from 1 to 283 months). The rates of complete response, partial response, and no response to cyclosporine and androgen treatment in RCC patients were 19.0%, 26.7%, and 54.3%, respectively. The 5- and 10-year prospective overall survival rates of RCC patients were 87.9% and 72.4%, respectively. The 5- and 10-year prospective clonal evolution rates were 15.3% and 20.0%, respectively. The 2-year prospective incidence of newly diagnosed karyotype abnormality after the initial diagnosis was 3.6%. The 5- and 10-year prospective leukemia transformation rates were 10.0% and 20.0%, respectively.
  
  
  CONCLUSIONS
  RCC shows clinical features similar to adult myelodysplastic syndrome. Children with RCC have a poor prognosis, an increased risk of transformation to leukemia, and a low response rate to cyclosporine treatment.
authors.affiliation: Pediatric Blood Diseases Centre, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin 300020, China. zhuxf6465@gmail.com.
authors.forename: Wen-Bin
authors.initials: WB
authors.lastname: An
authors.forename: Pei-Hong
authors.initials: PH
authors.lastname: Zhang
authors.forename: Yuan-Yuan
authors.initials: YY
authors.lastname: Ren
authors.forename: Ye
authors.initials: Y
authors.lastname: Guo
authors.forename: Shu-Chun
authors.initials: SC
authors.lastname: Wang
authors.forename: Xiao-Juan
authors.initials: XJ
authors.lastname: Chen
authors.forename: Wen-Yu
authors.initials: WY
authors.lastname: Yang
authors.forename: Li-Xian
authors.initials: LX
authors.lastname: Chang
authors.forename: Xiao-Fan
authors.initials: XF
authors.lastname: Zhu
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D060965:Clonal Evolution; D005260:Female; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D009190:Myelodysplastic Syndromes / Q000188:drug therapy* / Q000401:mortality; D010198:Pancytopenia / Q000188:drug therapy* / Q000401:mortality; D011379:Prognosis; D012189:Retrospective Studies