[Effectiveness of immunosuppressive therapy for childhood aplastic anemia and its predictive factors].
OBJECTIVE
To evaluate the effectiveness of immunosuppressive therapy (IST) for childhood aplastic anemia (AA) and its predictive factors.


METHODS
The medical data of 110 children with AA who received IST between February 2003 and November 2009 were retrospectively studied. Of these patients, 83 were diagnosed as severe AA (SAA) and 27 were non-SAA. The former group was treated with antithymocyte globulin (ATG) + ciclosporin (CSA) +prednisone + androgens and the latter with CSA + prednisone + androgens.


RESULTS
Total response rates in the SAA and non-SAA groups were 69.9% and 70.4%, respectively. Univariate analysis showed that disease duration, CD34+ cell level in bone marrow (BM) and the expression of CD4+CD25+ regulatory T cell in BM were related to the severity of disease but not correlated with treatment response. Age, disease duration and proportions of CD3+ and CD8+ T cells in BM were predictive factors for response to IST. Multivariate analysis revealed that patients aged more than 10 years and patients with a CD8+ cell proportion in BM of over 25% had hazard ratios of 3.36 and 3.59 for treatment failure respectively.


CONCLUSIONS
IST is effective in the treatment of childhood AA. Age, disease duration and the proportion of CD8+ T cell in BM are predictive factors for response to IST.
Division of Hematology-oncology, Children's Hospital of Zhejiang University School of Medicine, Hangzhou 310003, China.
Fen-Ying
FY
Zhao
Xiao-Jun
XJ
Xu
Hua
H
Song
Shi-Long
SL
Yang
Yong-Min
YM
Tang
D000293:Adolescent; D000741:Anemia, Aplastic / Q000188:drug therapy* / Q000276:immunology; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D008297:Male; D012189:Retrospective Studies
D007166:Immunosuppressive Agents