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[Treatment outcomes of 125 children with aplastic anemia]
Cheng-Juan Luo1, Jing Chen, Hui-Liang Xue
1Shanghai Jiao Tong University, School of Medicine Shanghai Children's Medical Center, Hematology/Oncology department, Shanghai 200127, China.
Objective:
To analyze the outcome of childhood aplastic anemia received allogenic hematopoietic stem cell transplantation (HSCT) and immunosuppressive therapy (IST).
Methods:
The clinical data of 125 consecutive children with aplastic anemia (AA) in our hospital were retrospectively analyzed.
Results:
According to the clinical manifestations, the 125 AA children were divided into two groups: SAA (n = 79) and NSAA (n = 46). There was no significant difference between the two groups in sex, age and follow-up duration (P > 0.05). The median follow-up was 25 (6 - 89) months. 103 cases received IST and 22 received allogenic HSCT. In SAA group, the response rate was better in patients received allogenic HSCT (n = 21) than in those received IST (n = 58) (85.7% vs 53.4%, P < 0.01). SAA patients received IST were further divided into two groups: 47 received antithymocyte globulin (ATG) and cyclosporine-A (CsA) combined therapy, 11 received CsA alone. There was no significant difference in total response rates (55.3% vs 45.5%, P = 0.555) and cure rates (42.6% vs 27.3%, P = 0.499) between the two groups. In NSAA group, 45 patients received IST and 1 received allogenic HSCT. In the IST treated NSAA patients, there was also no statistic significance in cure rates (36.4% vs 32.4%, P = 0.806) and total effective rates (63.6% vs 64.7%, P = 0.949) between ATG and CsA combined therapy (n = 11) and CsA alone therapy (n = 34).
Conclusion:
The outcome of children with AA received allogenic HSCT was obviously better than those received IST. IST is still the choice for patients without suitable donors for HSCT.
