[Treatment outcomes of 125 children with aplastic anemia].
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).


CONCLUSIONS
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.
Shanghai Jiao Tong University, School of Medicine Shanghai Children's Medical Center, Hematology/Oncology department, Shanghai 200127, China.
Cheng-Juan
CJ
Luo
Jing
J
Chen
Hui-Liang
HL
Xue
Jing-Yan
JY
Tang
Ci
C
Pan
Hua
H
Jiang
Lu
L
Dong
Chang-Ying
CY
Luo
Qi-Dong
QD
Ye
Min
M
Zhou
Long-Jun
LJ
Gu
D000741:Anemia, Aplastic / Q000628:therapy; D000961:Antilymphocyte Serum / Q000627:therapeutic use; D002648:Child; D016572:Cyclosporine / Q000627:therapeutic use; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D016896:Treatment Outcome
D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine