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First-line immunosuppressive therapy with rATG and CsA for severe aplastic anemia: 15 years’ experience

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Abstract

Rabbit antithymocyte globulin (rATG) instead of horse ATG has been used for severe aplastic anemia (SAA) patients in China. This study aimed to investigate the hematologic responses and long-term overall survival (OS) outcomes in SAA patients who received rATG and cyclosporine as first-line immunosuppressive therapy. We analyzed data of 542 SAA patients treated with this therapy between 2005 and 2019. The median age was 20 (range, 2–80) years, and the median follow-up time was 45.5 (range, 0.1–191.4) months. The early mortality rate was 3.9%. The overall response rates (ORRs) were 40.2%, 56.1%, and 62.4% at 3, 6, and 12 months, respectively. The 6- and 12-month ORR of patients treated with 3 mg/kg/d of rATG in 2015–2019 seemed higher than that of patients treated with 3.5–3.75 mg/kg/day in 2005–2014 (60.2% vs. 54.9%, P = 0.30 and 69.9% vs. 60.1%, P = 0.049, respectively). The 10-year cumulative incidences of relapse and clonal evolution were 10.6 ± 2.9% and 7.5 ± 1.5%, respectively. The 10-year OS rate and event-free survival rate were 80.1 ± 2.1% and 75.6 ± 3.7%, respectively. Age, disease severity, treatment periods, and the interval from diagnosis to IST were independent predictors of OS. In conclusion, 3 mg/kg/day rATG is effective as first-line treatment for SAA.

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Funding

This study was supported by Grant 81900127 of National Natural Science Foundation of China.

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Authors and Affiliations

  1. Anemia Therapeutic Centre, 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

    Jing Hu, Li Zhang, Xin Zhao, Xu Liu, Liping Jing, Kang Zhou, Yuan Li, Yang Li, Jianping Li, Lei Ye, Guangxin Peng, Huihui Fan, Wenrui Yang, Yang Yang, Youzhen Xiong, Lin Song & Fengkui Zhang

Authors
  1. Jing Hu
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  2. Li Zhang
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  3. Xin Zhao
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  4. Xu Liu
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  5. Liping Jing
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  6. Kang Zhou
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  7. Yuan Li
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  16. Lin Song
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  17. Fengkui Zhang
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Contributions

Fengkui Zhang served as the principal investigator for this study. Jing Hu analyzed the data and drafted the manuscript. Xin Zhao, Li Zhang, and Fengkui Zhang interpreted data and revised the manuscript. Xu Liu contributed to Data collection. Liping Jing, Kang Zhou, Yuan Li, Yang Li, Jianping Li, Lei Ye, Guangxin Peng, Huihui Fan, Wenrui Yang, Yang Yang, Youzhen Xiong, and Lin Song contributed to patient recruitment and treatment.

Corresponding author

Correspondence to Fengkui Zhang.

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This study was approved by the ethics committee of Blood Diseases Hospital, CAMS, and PUMC. All procedures followed were in accordance with the Helsinki Declaration of 1975, as revised in 2008.

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Written informed consent was obtained for each patient.

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The authors declare no conflict of interest.

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Hu, J., Zhang, L., Zhao, X. et al. First-line immunosuppressive therapy with rATG and CsA for severe aplastic anemia: 15 years’ experience. Ann Hematol 101, 2405–2412 (2022). https://doi.org/10.1007/s00277-022-04952-2

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