Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, People's Republic of China.
Department of Hematology, Affiliated Cancer Hospital of Zhengzhou University, Henan Cancer Hospital, Institute of Hematology of Henan Province, Zhengzhou, People's Republic of China.
Drug Des Devel Ther. 2020 Dec 30;14:5711-5719. doi: 10.2147/DDDT.S275975. eCollection 2020.
For symptomatic non-severe aplastic anemia (NSAA) patients who cannot afford anti-thymocyte globulin (ATG) or allogeneic hematopoietic stem cell transplantation (HSCT), tacrolimus (FK) may be an option if these patients do not respond or become tolerant to cyclosporine A (CsA).
We enrolled 101 NSAA patients who were refractory or intolerant to CsA with no chance of HSCT or ATG treatment and treated these patients with tacrolimus for at least 6 months, with follow-up for at least one year.
The overall response rate (ORR) was 38.6% (complete response: 9.9%; partial response: 28.7%), and the median time to optimal response was 6 (3～10) months. Thirty-two (31.7%) cases had elevated creatinine levels. Eight (7.9%) cases had elevations in AST/ALT. A total of 25.6% (10/39) of patients relapsed at the end of follow-up. Age (=0.0005), FK concentration (4.0~12 ng/mL, =0.0005) and intolerance to CsA (=0.012) were the independent risk factors for ORR. Treg cell levels pre-FK treatment were much lower than those of healthy controls (3.7±0.6% vs 6.8±0.7%, =0.0004) but increased significantly after FK treatment (3.7±0.6% vs 7.1±0.8%, =0.0039).
Our data suggest that tacrolimus is a salvage treatment for patients with NSAA that is refractory or intolerant to CsA.
对于因经济原因无法负担抗胸腺细胞球蛋白（ATG）或异基因造血干细胞移植（HSCT）的症状性非重型再生障碍性贫血（NSAA）患者，如果这些患者对环孢素 A（CsA）无反应或耐受，则他克莫司（FK）可能是一种选择。
我们纳入了 101 例对 CsA 无反应或不耐受且无 HSCT 或 ATG 治疗机会的 NSAA 患者，这些患者至少接受了 6 个月的 FK 治疗，并进行了至少 1 年的随访。
总体反应率（ORR）为 38.6%（完全缓解：9.9%；部分缓解：28.7%），最佳反应时间中位数为 6（3～10）个月。32 例（31.7%）患者的肌酐水平升高。8 例（7.9%）患者的 AST/ALT 升高。共有 25.6%（10/39）的患者在随访结束时复发。年龄（=0.0005）、FK 浓度（4.0～12ng/ml，=0.0005）和对 CsA 的不耐受（=0.012）是 ORR 的独立危险因素。FK 治疗前 Treg 细胞水平明显低于健康对照组（3.7±0.6% vs 6.8±0.7%，=0.0004），但 FK 治疗后明显升高（3.7±0.6% vs 7.1±0.8%，=0.0039）。
我们的数据表明，FK 是对 CsA 无反应或不耐受的 NSAA 患者的一种挽救治疗方法。
