CASE REPORT article
Front. Immunol.
Sec. Autoimmune and Autoinflammatory Disorders : Autoimmune Disorders
Volume 16 - 2025 | doi: 10.3389/fimmu.2025.1609771
Rapid Early Remission in a Patient with Severe Aplastic Anemia: A Case Report of Hetrombopag, Cyclosporine, and Danazol Combination Therapy
Provisionally accepted
- First Affiliated Hospital of Jilin University, Changchun, Jilin Province, China
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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.
Keywords: Severe aplastic anemia, case report, Immunosupressive agents, antithymocyte globulin, Cyclosporine
Received: 10 Apr 2025; Accepted: 04 Aug 2025.
Copyright: © 2025 Dai, Guo, Guo and Liu. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Qiuju Liu, First Affiliated Hospital of Jilin University, Changchun, 130012, Jilin Province, China
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