病例报告：短程降纤肽联合艾库珠单抗治疗TA-TMA
Case Report: Short-course defibrotide combined with eculizumab for TA-TMA.
摘要(中文译文)
移植相关血栓性微血管病（TA-TMA）是造血干细胞移植后的严重并发症，其发病机制尚未完全阐明，目前缺乏高效的治疗方案。本文报道1例女性急性髓系白血病（AML）患者，在第二次异基因造血干细胞移植（allo-HSCT）后3个月诊断为TA-TMA。在及时停用环孢素（CSA），并经抗感染、糖皮质激素及血浆置换治疗无效后，采用短程联合方案——去纤苷（10天）联合依库珠单抗（3周）——进行治疗。TA-TMA相关指标迅速改善并恢复正常。随访期间，患者原发病持续缓解，且5个月内无TA-TMA复发。在第二次allo-HSCT后8个月，因并发肺炎和脓毒症导致TA-TMA复发。再次给予相同的短程联合方案——去纤苷（18天）联合依库珠单抗（3周）——治疗后，患者的临床和实验室指标再次改善。本研究首次报道了采用去纤苷联合依库珠单抗短程联合治疗TA-TMA。该联合方案显示出良好的疗效和耐受性，为难治性TA-TMA提供了一种潜在的治疗选择，但其更广泛的适用性仍需进一步研究和验证。
Abstract
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.
如何引用
AMA
Yan Yan, Xiaoning Li, Linyu Li, Shaoli Zhang, Liangming Ma, Lifang Huang, et al. Case Report: Short-course defibrotide combined with eculizumab for TA-TMA.. Frontiers in immunology. 2026; doi:10.3389/fimmu.2026.1771562.
APA
Yan Yan, Xiaoning Li, Linyu Li, Shaoli Zhang, Liangming Ma, Lifang Huang, et al (2026). Case Report: Short-course defibrotide combined with eculizumab for TA-TMA.. Frontiers in immunology. https://doi.org/10.3389/fimmu.2026.1771562
GB/T 7714
Yan Yan, Xiaoning Li, Linyu Li, Shaoli Zhang, Liangming Ma, Lifang Huang, et al. Case Report: Short-course defibrotide combined with eculizumab for TA-TMA.[J]. Frontiers in immunology, 2026 doi:10.3389/fimmu.2026.1771562.
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