OBJECTIVETo analyze the efficacy and side-effects of combination of rabbit antithymocyte globulin (ATG) and cyclosporine A (CsA) as the first-line immunosuppressive therapy (IST) for adult severe aplastic anemia (SAA) patients.

METHODSAdult SAA or very severe aplastic anemia (VSAA) patients treated with rabbit ATG + CsA as first line therapy in our hospital from 2003 to 2008 were retrospectively analysed and the therapeutic response relevant factors were analysed.

RESULTSSeventy-nine patients were enrolled. Of all these patients, 6 died within 3 months after IST. The overall response rate was 82.2% and the median time to transfusion independent was 60 days. The therapeutic response rate in 32 SAA patients (100%) was significantly higher than that in 41 VSAA cases (68.3%) (P = 0.001). Patients with neutrophil response to G-CSF treatment had a higher IST response rate than those without response to G-CSF (100% vs 67.5%, P = 0.001). Sixty-one patients (77.2%) occurred serum sickness reaction. Three patients relapsed and two developed clonal hematological abnormalities after IST. The 3-year overall survival for all the patients was 88.9%.

CONCLUSIONSRabbit ATG in combination with CsA as first-line IST for adult SAA can lead to excellent treatment outcomes with minor adverse effects.