Abstract
To assess the outcomes of children with acquired aplastic anemia (AA) treated in China with first-line porcine anti-lymphocyte immunoglobulin (p-ALG)/rabbit anti-thymocyte immunoglobulin (r-ATG) combined with cyclosporine A (CSA). We performed a single-center, non-randomized, retrospective cohort study to assess the outcomes of 189 children with AA treated in China with first-line p-ALG/r-ATG combined with CSA between 2014 and 2018. No significant differences were observed in the overall response rates at 3, 6, 12, or 24months (3months: 61.9% vs 67.4%, P=.5; 6months: 70.9% vs 73.9%, P=.69; 12months: 77.3% vs 73.3%, P=.58; 24months: 81.6% vs 78.6%, P=.59) after either p-ALG- or r-ATG-based immunosuppressive therapy. No significant differences were observed in overall survival or failure-free survival between the p-ALG group and the r-ATG group. Our results reveal that the therapeutic efficacy and safety of p-ALG combined with CSA did not differ significantly from those of r-ATG combined with CSA as first-line therapy for pediatric patients with AA. Moreover, p-ALG has the advantage of significantly lower cost compared with r-ATG.
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