The long-term outcome and safety of severe aplastic anemia treated with porcine antilymphocyte globulin plus cyclosporine, with or without thrombopoietin receptor agonists: a double-center retrospective study

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Objectives : To evaluate the long-term outcome and safety of porcine antilymphocyte globulin plus cyclosporine, with or without thrombopoietin receptor agonist (TPO-RA), for patients with severe aplastic anemia (SAA) and identification of factors influencing hematology response and long-term outcome. Methods : We retrospectively analyzed the data of 85 people with newly diagnosed SAA who underwent porcine antithymocyte globulin (p-ATG) plus cyclosporine (CsA), with or without TPO-RA in the second Xiangya hospital and the first people’s hospital of Chenzhou during 2014 to 2023. Results : The overall response rates were 55.3% and 65.9% with 3 and 6 months. Hematologic response rates for the TPO-RA group were 66.7% and 72.3%, at 3 and 6 months and without the TPO-RA group were 27.8% and 55.6%. In multivariate analysis, platelet count＞10x10 9 /L was a predictor of favorable response at 6 months (P= 0.015). The median follow-up time for all patients was 39 months (range 0.4~104), the 5-year OS rate of 90.6% [95% CI = 82.1–95.2%], and the FFS rate was 68.9% [95% CI =56.6–78.4%]. The very severe aplastic anemia（VSAA）subgroup was an independent unfavorable predictor of failure-free survival (FFS) and overall survival (OS). 12 patients (14.1%) suffered from serum sickness, and 9.5% of patients had mild hepatic impairment. Conclusions : p-ATG can be used as an appropriate alternative preparation for rabbit ATG, with fewer side effects and lower medical costs, and combined with TPO-RA may improve early hematologic response rates.