Abstract
The current standard first-line therapy for aplastic anemia (AA) consists of antithymocyte globulin (ATG), cyclosporine, and thrombopoietin receptor agonists (TPO-RAs). A subset of patients exhibit treatment intolerance or develop refractory/relapsed disease, for whom treatment options are limited. Umbilical cord blood exhibits immunomodulatory effects, regulates the bone marrow microenvironment, and promotes hematopoietic reconstitution, thereby demonstrating therapeutic potential for AA. In this prospective clinical study, cord blood infusion was administered in combination with cyclosporine and hetrombopag for the treatment of AA (n = 11). The primary endpoint was the hematologic response at 3 months post-treatment. By 3 months, 8 of 11 patients achieved at least one lineage hematologic response. By 6 months, 3 patients attained complete response (CR), 5 achieved partial response (PR), yielding an overall response rate (ORR) of 8/11. After a median follow-up of 23 months, 5 patients maintained sustained CR and 3 sustained PR, with the ORR remaining at 8/11. The median time to trilineage response was 112 days (range: 18-168 days) among 8 responsive patients. Two patients with SAA refractory to treatment succumbed to pulmonary infections and pneumorrhagia. No significant adverse reactions were observed in cord blood-treated patients. This small-scale study introduces a novel regimen combining umbilical cord blood infusion, cyclosporine, and hetrombopag for treating aplastic anemia. With a median follow-up of 23 months, the overall response rate reached 8/11 and the CR rate 5/11. The safety and efficacy of this regimen were preliminarily assessed, suggesting a potential therapeutic alternative for patients ineligible for standard immunosuppressive therapy.