| Summary: | Yali Du,1 Yuzhou Huang,1 Wenzhe Zhou,1 Xinjian Liu,2 Fangfei Chen,1 Chen Yang,1 Miao Chen,1 Jing Ruan,1 Bing Han1 1Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, People’s Republic of China; 2Department of Hematology, Affiliated Cancer Hospital of Zhengzhou University, Henan Cancer Hospital, Institute of Hematology of Henan Province, Zhengzhou, People’s Republic of ChinaCorrespondence: Bing HanDepartment of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, People’s Republic of ChinaEmail hanbing_li@sina.com.cnBackground: 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).Methods: 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.Results: 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 (P=0.0005), FK concentration (4.0∼ 12 ng/mL, P=0.0005) and intolerance to CsA (P=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%, P=0.0004) but increased significantly after FK treatment (3.7± 0.6% vs 7.1± 0.8%, P=0.0039).Conclusion: Our data suggest that tacrolimus is a salvage treatment for patients with NSAA that is refractory or intolerant to CsA.Keywords: non-severe aplastic anemia, tacrolimus, cyclosporine A
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