Hematopoietic stem cell transplantation for acquired aplastic anemia in 2024: Report from the Chinese Blood and Marrow Transplantation Registry Group (CBMTRG).

Allogeneic hematopoietic stem cell transplantation (HCT) is a curative treatment for acquired aplastic anemia (AA). More than 1,000 cases of transplants for AA are performed annually in China, and transplant protocols have been continuously refined over recent years. To characterize contemporary transplant practices in China and provide useful insights for global professionals, this study analyzed the 2024 Chinese National Registry data. In 2024, 1,919 transplants were reported among 1,893 patients by 158 centers. Haploidentical donor (HID) was the leading donor source (57.22%). Matched sibling donor (MSD) and unrelated donor (URD) were used in 19.23% and 17.87% of cases, respectively. The G-CSF/ATG-based platform (84.42%) was the predominant transplant modality, followed by the ATG/ATLG+PTCY platform (14.17%). Low-dose busulfan was frequently used in conditioning regimens for both platforms. Due to the dominant use of HID and peripheral blood stem cell grafts, novel graft-versus-host disease (GVHD) prophylactic strategies have been explored. Based on a representative contemporary cohort of 226 patients, we report a favorable 1-year survival rate of 92.0% and a GVHD-/failure-free survival rate of 77.8%, which are comparable to reports from the EBMT and CIBMTR. Moreover, modern HCT practice supports broader consideration of transplantation for older patients, especially those aged 40-50 years. In conclusion, our report offers a comprehensive overview of the current HCT landscape in China and serves as a valuable resource for global practice.