Case Report: Short-course defibrotide combined with eculizumab for TA-TMA

Transplantation-associated thrombotic microangiopathy (TA-TMA) is a severe complication following hematopoietic stem cell transplantation, with its pathogenesis not yet fully understood and a lack of highly effective treatment options currently available. This article reports a case of a female patient with acute myeloid leukemia (AML) who was diagnosed with TA-TMA three months after a second allogeneic hematopoietic stem cell transplantation (allo-HSCT). After promptly discontinuing cyclosporine (CSA), and following ineffective treatments with anti-infective therapy, glucocorticoids, and plasma exchange, a short-course combination regimen-defibrotide (10 days) and eculizumab (3 weeks)-was administered. The TA-TMA-related indicators rapidly improved and returned to normal. During follow-up, the patient's primary disease remained in remission, and she remained free of TA-TMA recurrence for five months. At eight months after the second allo-HSCT, TA-TMA recurred due to concurrent pneumonia and sepsis. The same short-course combination regimen-defibrotide (18 days) and eculizumab (3 weeks)-was administered, and the patient showed improvement in clinical and laboratory parameters again. This study is the first to report the use of a short-course combination therapy with defibrotide and eculizumab for TA-TMA. The combination regimen demonstrated good efficacy and tolerability, offering a potential treatment option for refractory TA-TMA, though its broader applicability requires further research and validation.