Unrelated donor hematopoietic stem cell transplantation compared to immunosuppressive therapy plus eltrombopag as first-line treatment for adults with severe aplastic anemia

作者：Liangliang Wu, Limin Liu, Xin Zhao, Ming Zhou, Andie Fu, Yuping Zhang, Wenrui Yang, Xiaowei Chen, Wenjian Mo, Caixia Wang, Yumiao Li, Shilin Xu, Shiyi Pan, Ruiqing Zhou, Fankai Meng, Fengkui Zhang, Depei Wu, Shunqing Wang · 发表于：Blood Cancer Journal · 年份：2024 · DOI：10.1038/s41408-024-01021-x · 被引用次数：9 · 研究领域：Hematopoietic Stem Cell Transplantation、Mesenchymal stem cell research、Polyomavirus and related diseases

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) and immunosuppressive therapy (IST) are the main therapeutic options for severe aplastic anemia (SAA) [ 1 ]. The first-line therapy for < 35-year-old adults with SAA is HLA-matched sibling donor (MSD) HSCT [ 1 ]. IST is the traditionally recommended first-line therapy for adult patients not eligible for MSD-HSCT [ 1 ]. Recently, eltrombopag (EPAG) plus IST as a front-line treatment improved the rate and rapidity of the hematologic response [ 2 ]. However, patients treated with IST + EPAG are at risk of severe infection, bleeding and clonal hematopoiesis [ 3 ]. Unrelated donor HSCT (URD-HSCT) for adults with SAA is currently recommended after IST failure [ 1 ]. Encouragingly, as transplantation technology advances, the survival outcomes of URD-HSCT have improved dramatically [ 4 , 5 ]. The above has stimulated the transplantation community to consider giving URD-HSCT a larger role as a first-line option for adult SAA patients who lack an MSD [ 4 , 5 , 6 , 7 , 8 ]. However, no studies have compared the outcomes of first-line URD-HSCT and IST + EPAG in adults.