Basic & Clinical Medicine ›› 2020, Vol. 40 ›› Issue (6): 759-764.

• Original Articles • Previous Articles     Next Articles

Comparison of intensive immunosuppressive therapy with haploidentical transplantation for young severe aplastic anemia

CHEN Miao1#, WU Ya-mei2#, ZHOU Dao-bin1, DUAN Ming-hui1, ZHUANG Jun-ling1, HAN Bing1*, WU Xiao-xiong2*   

  1. 1. Department of Hematology, Peking Union Medical College Hospital, CAMS & PUMC, Beijing 100730;
    2. Department of Hematology, the Fourth Medical Center of Chinese PLA General Hospital, Beijing 100048, China
  • Received:2019-12-17 Revised:2020-03-26 Online:2020-06-05 Published:2020-05-29
  • Contact: *hanbing_li@sina.com;xiongwuxiao@sohu.com

Abstract: Objective A retrospective study was performed to compare intensive immunosuppressive therapy (IST) and haploidentical donor hematopoietic stem cell transplantation (HID-HSCT) for young patients with severe aplastic anaemia(SAA). Methods Fifty-five SAA patients aged 14-30 years were included. The patients were treated with anti-thymocyte immunoglobulin(ATG) combined with cyclosporine A(CsA)(29 cases, IST group) or HID-HSCT(26 cases, HID-HSCT group). Results The complete response(CR) rates in the IST and HID-HSCT groups were 58.6% vs 84.6%(P=0.034), and the overall response(OR) rates were 86.2% vs 84.6%. No significant difference was found in estimated 5-year overall survival(IST 77.9%±11.7% vs HID-HSCT 82.1%±8.4%) and event-free survival(IST 67.6%±11.7% vs HID-HSCT 69.2%±9.1%). The relapse rate of the IST group was 3.4%, and the clonal transformation rate was 13.8%. In HID-HSCT group, the relapse and clonal transformation rates were both 0%, and the incidence rates of grades 2-4 aGVHD and cGVHD were 19.2% and 15.4% respectively. The hospitalization expenses were significantly less in the IST group than in that of HID-HSCT group(median 168 000 yuan vs 378 000 yuan, P<0.001). Conclusions IST shows similar OR, OS and EFS but much less expense comparing with that of HID-HSCT. IST remains an appropriate choice for young SAA patients without sibling donors.

Key words: aplastic anemia, intensive immunosuppressive therapy, donor transplantation

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