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[Clinical characteristics of clonal evolution after immunosuppressive therapy in children with severe/very severe

Jing-Liao Zhang1, Tian-Feng Liu, Li-Xian Chang

  • 1Department 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.

Zhongguo Dang Dai Er Ke Za Zhi = Chinese Journal of Contemporary Pediatrics
|January 20, 2017
PubMed

Insights

Children with severe/very severe aplastic anemia (SAA/VSAA) receiving immunosuppressive therapy (IST) face risks of clonal evolution. High CD3+ T cell percentage or lack of IST response predict unfavorable progressions, impacting survival outcomes.

Area of Science:

  • Hematology
  • Pediatric Oncology
  • Immunosuppression Therapy

Background:

  • Severe/very severe aplastic anemia (SAA/VSAA) is a life-threatening condition.
  • Immunosuppressive therapy (IST) is a standard treatment for SAA/VSAA.
  • Clonal evolution can occur after IST, leading to adverse outcomes.

Purpose of the Study:

  • To investigate the clinical characteristics of clonal evolution after IST in pediatric SAA/VSAA.
  • To identify risk factors associated with unfavorable clonal evolution post-IST.

Main Methods:

  • Retrospective analysis of clinical data from 231 children with newly-diagnosed SAA/VSAA treated with IST.
  • Evaluation of the incidence and risk factors for clonal evolution, including cytogenetic abnormalities and paroxysmal nocturnal hemoglobinuria (PNH) clones.
  • Univariate and Cox multivariate analyses to determine independent risk factors for unfavorable progressions.

Main Results:

  • The 5-year overall survival rate was 82.7%.
  • Fifteen patients experienced clonal evolution post-IST, with 5 favorable (PNH and trisomy 8) and 10 unfavorable (monosomy 7, MDS/AML) progressions.
  • Independent risk factors for unfavorable progressions included elevated CD3+ T cell percentage (>80%) and lack of response to IST.

Conclusions:

  • Children with SAA/VSAA and a high CD3+ T cell percentage at diagnosis are at increased risk of unfavorable progressions.
  • Lack of response to IST is a significant predictor of adverse outcomes in pediatric SAA/VSAA patients.
Abstract

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