Center for Pediatric Blood Disease, State Key Laboratory of Experimental Hematology, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China.
Department of Hematology and Oncology, Soochow University Affiliated Children's Hospital, Suzhou, China.
Acta Haematol. 2019;142(3):162-170. doi: 10.1159/000499065. Epub 2019 May 15.
Aplastic anemia (AA) is a hematologic disease characterized by pancytopenia and hypocellular bone marrow, potentially leading to chronic anemia, hemorrhage, and infection. The China Aplastic Anemia Committee and British Committee for Standards in Haematology guidelines recommend hematopoietic stem-cell transplantation (HSCT) or immunosuppressive therapy (IST) comprising antithymocyte globulin (ATG) with cyclosporine (CsA) as initial treatment for AA patients. With limited epidemiological data on the clinical management of AA in Asia, a prospective cohort registry study involving 22 AA treatment centers in China was conducted to describe the disease characteristics of newly diagnosed AA patients and investigate real-world treatment patterns and patient outcomes. Of 340 AA patients, 72.9, 12.6, and 3.5% were receiving IST, traditional Chinese medicine, and HSCT, respectively, at baseline; only 22.2% of IST-treated patients received guideline-recommended ATG with CsA initially. Almost all patients received supportive care (95.6%) as blood transfusion (97.8%), antibiotics (63.7%), and/or hematopoietic growth factors (58.2%). Overall, 64.8% achieved a partial or complete response, and 0.9% experienced relapse. No new safety concerns were identified; serious adverse events were largely unrelated to the treatment regimen. These results demonstrate the need to identify and minimize treatment barriers to standardize and align AA management in China with treatment guideline recommendations and further improve patient outcomes.
再生障碍性贫血(AA)是一种血液疾病,其特征为全血细胞减少和骨髓细胞减少,可能导致慢性贫血、出血和感染。中国再生障碍性贫血委员会和英国血液学标准委员会指南建议造血干细胞移植(HSCT)或包括抗胸腺细胞球蛋白(ATG)加环孢素(CsA)的免疫抑制疗法(IST)作为 AA 患者的初始治疗。由于亚洲关于 AA 临床管理的流行病学数据有限,因此进行了一项涉及中国 22 个 AA 治疗中心的前瞻性队列注册研究,以描述新诊断的 AA 患者的疾病特征,并调查真实世界的治疗模式和患者结局。在 340 名 AA 患者中,分别有 72.9%、12.6%和 3.5%在基线时接受 IST、中药和 HSCT;只有 22.2%接受 IST 治疗的患者最初接受了指南推荐的 ATG 加 CsA。几乎所有患者都接受了支持性治疗(95.6%),包括输血(97.8%)、抗生素(63.7%)和/或造血生长因子(58.2%)。总体而言,64.8%的患者获得了部分或完全缓解,0.9%的患者复发。没有发现新的安全问题;严重不良事件与治疗方案基本无关。这些结果表明,需要识别和减少治疗障碍,以标准化和调整中国的 AA 管理与治疗指南建议一致,并进一步改善患者结局。