OBJECTIVETo assess the short term curative efficacy and long-term survival outcomes of severe aplastic anemia patients following antithymocyte globulin/lymphoglobulin (ATG/ALG) with or without cyclosporine (CsA).

METHODSA total of 345 cases hospitalized in our hospital between December 1982 and June 2011 were enrolled into this study. We assessed the response rates 3 and 6 months after ATG/ALG, and estimated the overall survival (OS) by Kaplan-Meier method for this cohort of patients.

RESULTSThe cohort of 345 patients was routinely followed-up with a median follow-up of 44.0 (range, 0.5 - 244.0) months. The response rates at 3 and 6 months were 29.9% and 45.4%, respectively. The differences in response rates at both 3 (39.2% vs 19.6%, P < 0.01) and 6 months (55.6% vs 34.0%, P < 0.01) between 184 non-severe aplastic anemia (mSAA) and 161 very severe aplastic anemia (VSAA) were statistically significant. The response rates among the different ATG preparations were comparative; but 3-(10.6%) and 6-month (25.5%) responses produced by rATG-Fresenius were significantly inferior to those by rATG-Sangstat (36.6% and 56.6%, respectively) (all P < 0.01). The 5-year OS was 61.7% (95%CI 55.4% - 68.0%) for the entire cohort of patients, and 5-year OS for mSAA patients \[71.0% (95%CI 62.9% - 79.1%)\] was superior to that of VSAA patients \[50.4% (95%CI 40.1% - 60.7%), P < 0.01\]; but for the patients treated from 2007, the difference of OS in the last 5 years between VSAA and mSAA was not significant \[ 73.7% (95%CI 52.2% - 95.2%) vs 89.7% (95%CI 79.5% - 99.9%); P = 0.24\]. Our study also confirmed the superiority of ATG/ALG + CsA regimen \[64.8% (95%CI 57.9% - 71.7%)\] over ATG/ALG alone \[32.6% (95%CI 15.7% - 49.5%)\] with regard to 5-year OS (P < 0.01); but the addition of recombinant human granulocyte colony-stimulating factor (rhG-CSF) to ATG/ALG had no benefit in terms of OS. rATG-S produced significantly better 5-year OS \[66.1% (95%CI 55.8% - 76.4%)\] than rATG-F \[46.6% (95%CI 35.9% - 57.3%); P < 0.01\].

CONCLUSIONS(1) The outcome of mSAA was superior to that of VSAA, but the latter was markedly improved in the last 5 years; (2) rATG-F was inferior to rATG-S with regard to 5-year OS; (3) Immunosuppressive treatment with ATG/ALG plus CsA was more effective than ATG/ALG alone; (4) The addition of rhG-CSF to ATG/ALG had no benefit in terms of OS.