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

Wenrui Yang; Xu Liu; Xin Zhao; Li Zhang; Guangxin Peng; Lei Ye; Kang Zhou; Yuan Li; Jianping Li; Huihui Fan; Yang Yang; Youzhen Xiong; Liping Jing; Fengkui Zhang

doi:10.1177/20406207221146031

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

Ther Adv Hematol. 2023 Jan 12:14:20406207221146031. doi: 10.1177/20406207221146031. eCollection 2023.

Authors

Wenrui Yang^{1

2}, Xu Liu^{1

2}, Xin Zhao^{1

2}, Li Zhang^{1

2}, Guangxin Peng^{1

2}, Lei Ye^{1

2}, Kang Zhou^{1

2}, Yuan Li^{1

2}, Jianping Li^{1

2}, Huihui Fan^{1

2}, Yang Yang^{1

2}, Youzhen Xiong^{1

2}, Liping Jing³, Fengkui Zhang^{1

2}

Affiliations

¹ State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.
² Wenrui Yang, Xu Liu, Xin Zhao, Li Zhang, Guangxin Peng, Lei Ye, Kang Zhou, Yuan Li, Jianping Li, Huihui Fan, Yang Yang, Youzhen Xiong, Fengkui Zhang is also affiliated to Tianjin Institutes of Health Science, Tianjin, China.
³ State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China Tianjin Institutes of Health Science, Tianjin 301600, China.

Abstract

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.

Keywords: IST; aplastic anemia; hematologic response; p-ATG; survival.