State Key Laboratory of Experimental Hematology, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Science & Peking Union Medical College, 288 Nanjing Road, Tianjin, 300020, People's Republic of China.
Ann Hematol. 2020 Aug;99(8):1727-1734. doi: 10.1007/s00277-020-04153-9. Epub 2020 Jun 29.
Hematopoietic stem cell transplantation (HSCT) and immunosuppressive therapy (IST) with antithymocyte globulin (ATG) and cyclosporine (CsA) have been widely accepted as the standard first-line treatments for severe aplastic anemia (SAA). However, most of the patients with SAA had a slim chance to access these strategies in developing countries. Here, we reported 10-year results in a cohort of 232 patients with SAA who received a novel IST of CsA, levamisole, and danazol (CsA&LMS-based regimen). The cumulative incidence of response was 52.1% at 6 months, 66.4% at 12 months, and 77.1% at 24 months. The 10-year overall survival (OS) and failure-free survival was 60.2% and 48.3%, respectively. Positive predictors of OS in multivariate analysis were higher pretreatment ANC, younger age, higher pretreatment absolute reticulocyte count (ARC), and response within 6 months. The probability of CsA&LMS discontinuation was 50.2% at 10 years. With a slow CsA&LMS taper, the actuarial risk for relapse was only 9.5%. The cumulative incidence of MDS/AML was 8.2% at 10 years. The long-term follow-up information demonstrated that the CsA&LMS regimen could be a promising strategy for patients with SAA in developing countries.
造血干细胞移植（HSCT）和免疫抑制治疗（IST）联合抗胸腺细胞球蛋白（ATG）和环孢素（CsA）已被广泛接受为严重再生障碍性贫血（SAA）的标准一线治疗方法。然而，发展中国家的大多数 SAA 患者获得这些治疗方案的机会微乎其微。在此，我们报告了 232 例 SAA 患者接受 CsA、左旋咪唑和丹那唑新型 IST（CsA&LMS 方案）的 10 年结果。6 个月时的反应累积发生率为 52.1%，12 个月时为 66.4%，24 个月时为 77.1%。10 年总生存率（OS）和无失败生存率分别为 60.2%和 48.3%。多因素分析中 OS 的阳性预测因素为治疗前 ANC 较高、年龄较小、治疗前绝对网织红细胞计数（ARC）较高以及 6 个月内出现反应。10 年后 CsA&LMS 停药的概率为 50.2%。随着 CsA&LMS 逐渐减量，复发的实际风险仅为 9.5%。10 年后 MDS/AML 的累积发生率为 8.2%。长期随访资料表明，CsA&LMS 方案可能是发展中国家 SAA 患者的一种有前途的治疗策略。
