Bing Han

Introduction: Aplastic anemia (AA) is characterized by bone marrow failure and cytopenia. Eltrombopag (ELT) is effective and safe for treating refractory/relapsed AA; however, reports on the long-term outcomes of transfusion-dependent non-severe AA (TD-NSAA) are limited. Methods: Patients with TD-NSAA refractory to immunosuppressive therapy (IST) or relapsed after IST, treated with ELT alone, and followed up for at least 12 months were retrospectively enrolled. The baseline characteristics of patients, efficacy and adverse effects of ELT, and relapse and clone evolution rates after ELT were documented. Results: Of the 55 patients with TD-NSAA included, 24 (43.6%) were men. Median age at diagnosis was 46 (19–80) years. Twenty-four patients had relapsed TD-NSAA, and 31 patients had refractory TD-NSAA. During the median follow-up period of 28 (12–48) months, the overall and complete response rates at 3, 6, and 12 months of ELT treatment were 38.2, 60.0, and 52.7 and 9.1, 14.6, and 9.1%, respectively. After a median follow-up of 28 (12–48) months, 21.2% (7/33) of patients experienced relapse, with a median duration from ELT treatment to relapse of 14 (6–45) months. Conclusion: ELT was effective in patients with relapsed/refractory TD-NSAA, with tolerable adverse effects.

Background: For the symptomatic non-severe aplastic anemia (NSAA) patients who cannot afford anti-thymocyte globulin (ATG) or allogeneic hematopoietic stem cell transplantation (HSCT), tacrolimus (FK) was occasionally reported to be an option if they were not response or tolerant to the cyclosporine A (CsA). Method: We collected and analyzed respectively 101 NSAA patients refractory or intolerant to CsA with no chance of HSCT or ATG, and treated them with tacrolimus for at least 6 months and followed up for at least one year. Results: The overall response (ORR) was 38.6% (complete response: 9.9%; partial response: 28.7%) and the median time to optimal response was 6 (3～10) months. 32 (31.7%) cases had elevated creatinine level. 8 (7.9%) cases had elevation of AST/ALT. 25.6% (10/39) of patients relapsed at the end of follow-up. Age (P=0.0005), FK concentration (4.0~12ng/ml, P=0.0005) and intolerance to CsA (P=0.012) were the independent risk factors for ORR. Treg cells level pre-FK treatment was much lower than 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 provide a possibility of tacrolimus as a salvage treatment for patients with NSAA refractory or intolerant to CsA.