Center for HUS Prevention, Control and Management at the Pediatric Nephrology, Dialysis and Transplantation Unit, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
Center for HUS Prevention, Control and Management at the Nephrology, Dialysis and Kidney Transplantation Unit, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
Transplantation. 2019 Feb;103(2):e48-e51. doi: 10.1097/TP.0000000000002505.
Atypical hemolytic uremic syndrome (aHUS) is life-threatening condition particularly when complicating allograft hematopoietic stem cell transplant (HSCT). In the past, the outcome was very poor with the majority of patients reaching end-stage renal disease or dying with little or no chances of kidney transplant (KTx) due to the high risk of relapse. The availability of C5 inhibition has opened up significant therapeutic opportunities and has improved the outcome particularly if complement dysregulation (CD) is the underlying pathogenetic mechanism.
We describe a peculiar case of a girl with aHUS complicating HSCT and her subsequent successful KTx received from the same donor thus performed without immunosuppression but anti-C5 inhibition.
Soon after HSCT performed for acute lymphoblastic leukemia, the patient developed a TMA due to CD and reached end-stage renal disease. After 2 years on dialysis, the patient received a KTx from her father who was already the HSCT donor. Given the full chimerism, no immunosuppressive agent was prescribed except a short (2 days) course of steroids and eculizumab to prevent aHUS relapse. Nine months after the KTx, the patient is well with normal renal function, no immunosuppression and continues eculizumab prevention of aHUS (1 infusion every 21 days).
All patients with transplant-associated thrombotic microangiopathy should be screened for the causes of CD. C5 inhibition with eculizumab is an important therapeutic resource to manage this complication. When KTx is necessary, immunosuppression can be safely withhold in case of same donor for both grafts and documented full chimerism.
非典型溶血尿毒综合征（aHUS）是一种危及生命的疾病，尤其是在合并异基因造血干细胞移植（HSCT）时。过去，由于复发风险高，大多数患者会发展至终末期肾病或死亡，几乎没有进行肾移植（KTx）的机会。C5 抑制的出现为治疗提供了重要机会，特别是在补体失调（CD）是潜在发病机制的情况下，改善了预后。
我们描述了一例女孩在 HSCT 后并发 aHUS 的特殊病例，随后她从同一供体成功接受了 KTx，因此在没有免疫抑制但有抗 C5 抑制的情况下进行了移植。
在接受急性淋巴细胞白血病的 HSCT 后不久，患者因 CD 发生 TMA 并发展为终末期肾病。在透析 2 年后，患者从已经是 HSCT 供体的父亲那里接受了 KTx。由于完全嵌合，除了短期（2 天）使用类固醇和依库珠单抗预防 aHUS 复发外，未使用任何免疫抑制剂。在 KTx 后 9 个月，患者情况良好，肾功能正常，无需免疫抑制，并继续使用依库珠单抗预防 aHUS（每 21 天输注一次）。
所有移植相关血栓性微血管病患者均应筛查 CD 的病因。用依库珠单抗抑制 C5 是治疗这种并发症的重要治疗资源。当需要 KTx 时，如果两个移植物的供体相同且有明确的完全嵌合，则可以安全地不使用免疫抑制剂。
