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Because of relapse after horse ATG (hATG) therapy, rabbit ATG (rATG) would be a realistic alternative as second line immunosuppressive therapy (IST) in severe aplastic anemia (SAA) patients. We investigated whether intensified intravenous (IV) CsA therapy with rATG would increase the response of IST in SAA patients. Sixty-one of the 123 patients received IV CsA therapy with rATG during initial 2 weeks then changed to oral form (IV CsA group), while other 62 patients just received oral CsA therapy with rATG (oral CsA group). Hematologic response rates at 3 and 6 months were not different between IV CsA group and oral CsA group (p = 0.795, p = 0.079). However, CsA levels during initial 15 days were higher in response-achieved group than response-not-achieved group. Intensive IV CsA group maintained CsA level ≥300 ng/ml during 15 days had higher responses at 6 months than non-intensive IV CsA group and oral CsA group (p = 0.009, p = 0.021). Intensive IV CsA group (HR = 3.239, 95% CI = 1.095–8.997, p = 0.013) independently predicted favorable the hematologic response at 6 months of IST. Early intensified CsA therapy was important to achieve favorable outcomes in IST including rATG.

We analyzed the role of antibiotic prophylaxis during decitabine treatment for MDS. The primary endpoint was the incidence of febrile episodes. The total number of decitabine cycles given to 28 patients was 131, and febrile episodes occurred in 15 cycles (11.5%). Antibiotic prophylaxis was orally administered in 95 cycles (72.5%). Febrile episodes were significantly less frequent among patients who received antibiotic prophylaxis (7.4%) than in those without prophylaxis (22.2%) (P=0.017). In conclusion, antibiotic prophylaxis reduced the incidence of febrile episodes in patients who received decitabine treatment for MDS, especially at earlier cycles and in the presence of severe cytopenia.

We retrospectively investigated the incidence, risk factors, and outcomes of SOS (sinusoidal obstruction syndrome; previously veno-occlusive disease [VOD]) after allogeneic hematopoietic stem cell transplantation (alloHSCT) in aplastic anemia. Two hundred and sixty patients were included in the analysis. SOS developed in 7.3% (n=19/260) of patients. Classical Cy (200 mg/m(2))-ATG was the most common conditioning regimen (84.2%) in the SOS group. The SOS mortality rate was 4/19 (21.1%). Univariate analyses revealed that Cy 200 mg/m(2) conditioning (p=0.035), classical Cy-ATG conditioning (p=0.007), and horse ATG conditioning (p0.001) were significant risk factors for developing SOS. Multivariate analysis revealed that only horse ATG conditioning was a poor prognostic factor (HR=3.484; 95% CI 1.226-9.904; p=0.002). Rabbit ATG (HR 12.719; 95% CI 2.332-69.373; p=0.003) and weight gain10% (HR 35.655; 95% CI 2.208-575.805; p=0.012) were risk factors in the overall SOS group. Both rabbit ATG conditioning and weight gain of more than 10% were associated with poor overall survival with a median of 1.2 months (5Y survival rate, any risk factor vs. none: 74.6% vs. 0.0%; p0.001; Fig. 2) in the SOS group. In conclusion, SOS is a relatively rare (7.3%) but highly fatal (21.1%) acute complication of alloHSCT in AA, and the horse ATG conditioning regimen was a significant risk factor for developing SOS.