Dai Yuting, Guo Qiang, Guo Xiaoxiao, Liu Qiuju
Department of Hematology, Cancer Center, the First Hospital of Jilin University, Changchun, China.
Front Immunol. 2025 Aug 25;16:1609771. doi: 10.3389/fimmu.2025.1609771. eCollection 2025.
Severe aplastic anemia (SAA) is a life-threatening bone marrow failure syndrome that is caused primarily by immune-mediated destruction of hematopoietic stem cells. Traditional treatment relies on immunosuppressive therapy (IST) with antithymocyte globulin (ATG) and cyclosporine (CSA). However, the toxicity and limited availability of ATG have spurred interest in ATG-free regimens. This case report describes a 28-year-old male with SAA who was treated with a combination of CSA, danazol, and hetrombopag (HPAG). The patient presented with pancytopenia and a hypocellular bone marrow, thus meeting the SAA criteria. He received CSA (5 mg/kg/day), HPAG (started at 10 mg and increased to 15 mg/day), and danazol (400 mg/day). Hematologic assessments using the NIH criteria revealed a partial response at 3 months and a complete response at 6 months, with reduced proportions of active T-cell subclones and no severe adverse events. This case suggests that the combination of CSA, HPAG, and danazol is effective in treating SAA, and a large-scale clinical trial is warranted to further confirm these promising results.
重型再生障碍性贫血（SAA）是一种危及生命的骨髓衰竭综合征，主要由免疫介导的造血干细胞破坏引起。传统治疗依赖于使用抗胸腺细胞球蛋白（ATG）和环孢素（CSA）的免疫抑制疗法（IST）。然而，ATG的毒性和可用性有限引发了对无ATG方案的兴趣。本病例报告描述了一名28岁的SAA男性患者，他接受了CSA、达那唑和海曲泊帕（HPAG）联合治疗。该患者表现为全血细胞减少和骨髓细胞减少，符合SAA标准。他接受了CSA（5mg/kg/天）、HPAG（起始剂量为10mg，增加至15mg/天）和达那唑（400mg/天）治疗。使用美国国立卫生研究院（NIH）标准进行的血液学评估显示，3个月时出现部分缓解，6个月时出现完全缓解，活性T细胞亚克隆比例降低，且无严重不良事件。该病例表明，CSA、HPAG和达那唑联合治疗SAA有效，有必要进行大规模临床试验以进一步证实这些有前景的结果。
