[Combination of rabbit antithymocyte globulin plus cyclosporin A as first-line immunosuppressive therapy for the childhood with severe aplastic anemia.].
OBJECTIVE
To analyse the efficacy and side-effects of rabbit antithymocyte globulin (ATG) and cyclosporin A (CsA) as the first-line therapy for childhood severe aplastic anemia (SAA).


METHODS
Seventy-one childhood SAA patients treated with rabbit ATG + CsA as first line therapy were retrospectively analysed.


RESULTS
Seventy-one SAA patients, including 38 SAA and 33 very severe aplastic anemia (VSAA), were enrolled. The median age was 12 years. Of these patients, 3 died within 3 months after the immunosuppressive therapy (IST). The overall response rate was 67.6% (46/68) and the median time to transfusion independent was 53 days. Thirty-three patients (48.5%) obtained remission in 3 months after the IST and 45 (67.2%) in 6 months. The response rates were 57.7% (15/26), 56.5% (13/23) and 94.7% (18/19) for patients less than 10 years old, 10 - 15 year-old and 15 - 18 year-old, respectively. Sixty patients suffered from serum sickness on the IST. Three patients relapsed and another 3 unrespond patients received retreatment of IST, and one patient progressed to myelodysplastic syndromes (MDS).


CONCLUSIONS
Rabbit ATG in combination with CsA as first line therapy for childhood SAA/VSAA can lead to overall response rate of 67.6% with minor adverse effects.
Liu
Li-Yuan
LY
Institute of Hematology and Blood Diseases Hospital, CAMS&PUMC, Tianjin 300020, China.
Wang
Hui-Jun
HJ
Zhang
Li
L
Jing
Li-Ping
LP
Zhou
Kang
K
Yang
Dong-Lin
DL
Li
Hong-Qiang
HQ
Liu
Qing-Guo
QG
Yan
Zhang-Song
ZS
Liu
Yong-Ze
YZ
Wu
Yu-Hong
YH
Chu
Yu-Lin
YL
Zhang
Feng-Kui
FK
D000741:Anemia, Aplastic / Q000628:therapy; D000818:Animals; D000961:Antilymphocyte Serum; D016572:Cyclosporine / Q000627:therapeutic use; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D011817:Rabbits; D016896:Treatment Outcome
D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine