Unrelated donor hematopoietic stem cell transplantation compared to immunosuppressive therapy plus eltrombopag as first-line treatment for adults with severe aplastic anemia
Year: 2024
Type: article
Abstract: 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.
Source: Blood Cancer Journal
Institutions: Guangzhou First People's Hospital, South China University of Technology, First Affiliated Hospital of Soochow University, Chinese Academy of Medical Sciences & Peking Union Medical College, Institute of Hematology & Blood Diseases Hospital, State Key Laboratory of Experimental Hematology, Tongji Hospital, Huazhong University of Science and Technology
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $4,990
Funders: National Natural Science Foundation of China, Government of Jiangsu Province, Huazhong University of Science and Technology, Natural Science Foundation of Guangdong Province, Tongji University, South China University of Technology, Chinese Academy of Medical Sciences, Soochow University, Peking Union Medical College, State Key Laboratory of Experimental Hematology, Priority Academic Program Development of Jiangsu Higher Education Institutions, Guangzhou First People's Hospital, Guangzhou Municipal Science and Technology Project, Tongji Medical College, Huazhong University of Science and Technology, Innovation Capability Development Project of Jiangsu Province
OpenAlex ID: W4392514706