OBJECTIVETo evaluate the efficacy of porcine anti-human lymphocyte globulin (P-ALG) plus cyclosporine A (CsA) therapy for severe aplastic anemia (SAA).

METHODSForty-eight SAA patients (31 males, 17 females) including 17 very severe aplastic anemias (vSAA) were treated with ALG plus CsA between 1999 to 2009 in our hospital and the outcomes were analyzed retrospectively for early mortality, response rate and quality, survival rate, toxicity and complications.

RESULTSThe median age was 28 (13 - 64) years. The interval from diagnosis to treatment was 45 days. The median neutrophil count at diagnosis was 0.178 × 10(9)/L. Overall response was 83.3% (54.2% complete, 29.2% partial) with a median time of 90 (23 - 380) days. 10.4% died of infection within 30 days mainly of fungi infection. Only 1 patient relapsed 2 years after treatment. No clonal disease was found. The 1.5-year survival rate was 87.5%. vSAAs had less response, higher early mortality and less survival (64.7%, 29.4% and 51.8%, respectively) compared to that of SAA (93.5%, 0, 100%, respectively, P < 0.05). Grouped patients with different age, gender, intervals between diagnosis and treatment and pre-existing infections had similar response. The main side effects were fever and skin rash (52.1%), serum sickness (16.7%), impaired liver function (60.4%) and hemorrhage (2.1%). No treatment-related mortality was found.

CONCLUSIONP-ALG plus CsA is an ideal and well tolerated treatment for SAA but not for vSAA.