Yang Wenrui, Liu Xu, Zhao Xin, Zhang Li, Peng Guangxin, Ye Lei, Zhou Kang, Li Yuan, Li Jianping, Fan Huihui, Yang Yang, Xiong Youzhen, Jing Liping, Zhang Fengkui
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.
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.
Ther Adv Hematol. 2023 Jan 12;14:20406207221146031. doi: 10.1177/20406207221146031. eCollection 2023.
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.
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.
This is a single-center retrospective study of medical records.
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.
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.
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.
自2009年以来，抗人T淋巴细胞猪免疫球蛋白（p-ATG）一直是中国重型再生障碍性贫血（SAA）患者免疫抑制治疗（IST）中最常用的抗胸腺细胞球蛋白制剂。
本研究旨在评估2010年至2019年接受p-ATG加环孢素（CsA）作为一线治疗的一大群SAA患者的早期血液学反应和长期预后。
这是一项对病历的单中心回顾性研究。
我们分析了2010年至2019年在我科接受p-ATG联合CsA作为一线IST治疗的1023例连续性获得性再生障碍性贫血（AA）患者的数据。
患者的中位年龄为24（4-75）岁，中位随访时间为57.2个月（3天-137.5个月）。早期死亡率为2.8%，中位死亡时间为0.9个月（3天-2.9个月）。3个月和6个月时的总缓解率分别为40.6%和56.1%。5年复发和克隆演变的累积发生率分别为9.0%[95%置信区间（CI）=4.2-16.0%]和4.5%（95%CI=1.4-10.6%）。5年总生存率（OS）和无事件生存率分别为83.7%（95%CI=81.1-86.0%）和50.4%（95%CI=47.1-53.5%）。
p-ATG联合CsA治疗AA有效且安全，在无法获得h-ATG的地区，p-ATG可作为标准IST方案的替代抗胸腺细胞球蛋白制剂。
