Antihuman T lymphocyte porcine immunoglobulin combined with cyclosporine as first-line immunosuppressive therapy for severe aplastic anemia in China: a large single-center, 10-year retrospective study

Background: Antihuman T lymphocyte porcine immunoglobulin (p-ATG) has been the most common ATG preparation in immunosuppressive therapy (IST) in Chinese patients with severe aplastic anemia (SAA) since 2009. Objectives: This study aimed to evaluate the early hematologic response and long-term outcomes of a large cohort of patients with SAA who received p-ATG plus cyclosporine (CsA) as first-line therapy from 2010 to 2019. Design: This is a single-center retrospective study of medical records. Methods: We analyzed the data of 1023 consecutive patients with acquired aplastic anemia (AA) who underwent p-ATG combined with CsA as a first-line IST treatment from 2010 to 2019 at our department. Results: The median age of the patients was 24 (4–75) years, and the median follow-up time was 57.2 months (3 days–137.5 months). There was an early mortality rate of 2.8% with a median death time of 0.9 months (3 days–2.9 months). The overall response rates were 40.6% and 56.1% at 3 and 6 months, respectively. The 5-year cumulative incidences of relapse and clonal evolution were 9.0% [95% confidence interval (CI) = 4.2–16.0%] and 4.5% (95% CI = 1.4–10.6%), respectively. The 5-year overall survival (OS) and event-free survival rates were 83.7% (95% CI = 81.1–86.0%) and 50.4% (95% CI = 47.1–53.5%), respectively. Conclusion: p-ATG combined with CsA for the treatment of AA is effective and safe, and p-ATG can be used as an alternative ATG preparation for the standard IST regimen in areas in which h-ATG is not available.