Journal
ANNALS OF HEMATOLOGY
Volume 100, Issue 1, Pages 53-61Publisher
SPRINGER
DOI: 10.1007/s00277-020-04296-9
Keywords
Antithymocyte globulin; Child; Immunosuppressive therapy; Predictor; Severe aplastic anemia
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Funding
- AiYou Foundation
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This retrospective study analyzed the clinical outcomes of 172 children with newly diagnosed SAA treated with rabbit ATG and CsA, and found that mild disease severity was the only predictor for favorable response at 6 months. Factors such as BFU-E levels and time interval before treatment were associated with overall survival rates.
This study retrospectively analyzed the clinical outcome of 172 children with newly diagnosed severe aplastic anemia (SAA) between January 2008 and April 2018, who received rabbit antithymocyte globulin (ATG) and cyclosporine (CsA) as first-line treatment. The median age at diagnosis was 5 years (range, 1-14). The overall response rates were 22.7%, 45.3%, and 61% at 40 days, 3 months, and 6 months, respectively, after rabbit ATG. In multivariate analysis, mild disease severity was the only predictor of favorable response at 6 months (P = 0.006). In the present study, median follow-up period was 63 months (range, 1-135). The 5-year overall survival (OS) and failure-free survival (FFS) rates were 90.5% and 70.4%. Multivariate analysis showed that erythroid burst-forming units (BFU-E) > 2/10(5)bone marrow mononuclear cell (BMMNC) (P = 0.037) and time interval before IST <= 30 days (P = 0.017) were independent positive predictors for OS, meanwhile BFU-E > 2/10(5)BMMNC (P = 0.029) was the only favorable prognostic factor for FFS.
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