title: [Clinical characteristics of clonal evolution after immunosuppressive therapy in children with severe/very severe aplastic anemia].
abstract: OBJECTIVE
  To evaluate the clinical characteristics and risk factors of clonal evolution after immunosuppressive therapy (IST) in children with severe/very severe aplastic anemia (SAA/VSAA).
  
  
  METHODS
  The clinical data of 231 children with newly-diagnosed SAA/VSAA who received IST were retrospectively studied. The incidence and risk factors of clonal evolution after IST were analyzed.
  
  
  RESULTS
  The 5-year overall survival rate of the 231 patients was 82.7%. Except for 18 cases of early deaths, 213 patients were evaluated for IST efficacy. Among the 231 patients, cytogenetic abnormalities for at least two chromosome metaphase were detectable in 14 (7.4%) patients, and PNH clones were detectable in either peripheral red blood cells or neutrophils for 95 patients. Among the 213 patients evaluated for IST efficacy, 15 patients experienced clonal evolution after IST. Five patients had PNH and trisomy 8 which were defined as favorable progressions, and ten patients experienced monosomy 7 and MDS/AML as unfavorable progressions. The 5-year accumulative incidence of favorable and unfavorable progression were (2.2±2.2)% and (4.8±3.3)%, respectively. Until the last follow-up, 100% (5/5) of patients with favorable progressions and 50% (5/10) of patients with unfavorable progressions survived. WBC>3.5×109/L, CD3+T cell percentage>80%, dosage of antithymocyte globulin >3.0 mg/(kg·d) and no response to IST were related to unfavorable progressions by univariate analysis. Cox multivariate analysis revealed that an increased CD3+T cell percentage (>80%) and no response to IST were independent risk factors for unfavorable progressions.
  
  
  CONCLUSIONS
  The children with SAA/VSAA who have an increased CD3+T cell percentage at diagnosis or have no response to IST are in high risks of unfavorable progressions.
authors.affiliation: Department of Pediatric Blood Diseases, Institute of Hematology & Blood Disease Hospotial, Chinese Academy of Medical Science & Peking Union Medical College, Tianjin 300020, China. xfzhu@ihcams.ac.cn.
authors.forename: Jing-Liao
authors.initials: JL
authors.lastname: Zhang
authors.forename: Tian-Feng
authors.initials: TF
authors.lastname: Liu
authors.forename: Li-Xian
authors.initials: LX
authors.lastname: Chang
authors.forename: Xia
authors.initials: X
authors.lastname: Chen
authors.forename: Yuan-Yuan
authors.initials: YY
authors.lastname: Ren
authors.forename: Cong-Cong
authors.initials: CC
authors.lastname: Sun
authors.forename: Chao
authors.initials: C
authors.lastname: Liu
authors.forename: Wen-Bin
authors.initials: WB
authors.lastname: An
authors.forename: Yang
authors.initials: Y
authors.lastname: Wan
authors.forename: Xiao-Juan
authors.initials: XJ
authors.lastname: Chen
authors.forename: Wen-Yu
authors.initials: WY
authors.lastname: Yang
authors.forename: Shu-Chun
authors.initials: SC
authors.lastname: Wang
authors.forename: Ye
authors.initials: Y
authors.lastname: Guo
authors.forename: Yao
authors.initials: Y
authors.lastname: Zou
authors.forename: Yu-Mei
authors.initials: YM
authors.lastname: Chen
authors.forename: Xiao-Fan
authors.initials: XF
authors.lastname: Zhu
mesh_terms: D000293:Adolescent; D000741:Anemia, Aplastic / Q000188:drug therapy* / Q000235:genetics / Q000276:immunology / Q000401:mortality; D002648:Child; D002675:Child, Preschool; D002869:Chromosome Aberrations; D060965:Clonal Evolution; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D008297:Male; D016016:Proportional Hazards Models; D012189:Retrospective Studies
chemical_list: D007166:Immunosuppressive Agents