Pediatric Nephrology, Dialysis and Transplantation Unit, Center for HUS Control, Prevention and Management, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, V. Commenda, 9, 20122, Milan, Italy.
Nephrology Unit, Center for HUS Prevention, Control and Management, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
J Nephrol. 2021 Dec;34(6):2027-2036. doi: 10.1007/s40620-021-01045-7. Epub 2021 May 6.
Patients with atypical hemolytic uremic syndrome (aHUS) have long been considered ineligible for kidney transplantation (KTx) in several centers due to the high risk of disease recurrence, graft loss and life-threatening complications. The availability of Eculizumab (ECU) has now overcome this problem. However, the best approach towards timing, maintenance schedule, the possibility of discontinuation and patient monitoring has not yet been clearly established.
This is a single center case series presenting our experience with KTx in aHUS.
This study included 26 patients (16 females) with a diagnosis of aHUS, who spent a median of 5.5 years on kidney replacement therapy before undergoing KTx. We compared the aHUS relapse rate in three groups of patients who underwent KTx: patients who received no prophylaxis, patients who underwent plasma exchange, those who received Eculizumab prophylaxis. Complement factor H-related disease was by far the most frequent etiology (n = 19 patients).
Untreated patients and patients undergoing pre-KTx plasma exchange prophylaxis had a relapse rate of 0.81 (CI 0.30-1.76) and 3.1 (CI 0.64-9.16) events per 10 years cumulative observation, respectively, as opposed to 0 events among patients receiving Eculizumab prophylaxis. The time between Eculizumab doses was tailored based on classic complement pathway activity (target to < 30%). Using this strategy, 12 patients are currently receiving Eculizumab every 28 days, 5 every 24-25 days, and 3 every 21 days.
Our experience supports the prophylactic use of Eculizumab in patients with a previous history of aHUS undergoing KTx, especially when complement dysregulation is well documented by molecular biology.
由于疾病复发、移植物丢失和危及生命的并发症风险高，长期以来，一些中心的不典型溶血性尿毒症综合征（aHUS）患者被认为不适合进行肾移植（KTx）。Eculizumab（ECU）的出现解决了这个问题。然而，关于时机、维持方案、停药的可能性以及患者监测等最佳方法尚未明确。
这是一项单中心病例系列研究，介绍了我们在 aHUS 患者中进行 KTx 的经验。
本研究纳入了 26 名（16 名女性）诊断为 aHUS 的患者，这些患者在接受 KTx 前平均接受了 5.5 年的肾脏替代治疗。我们比较了三组接受 KTx 的患者的 aHUS 复发率：未接受预防治疗的患者、接受血浆置换的患者、接受 Eculizumab 预防治疗的患者。补体因子 H 相关疾病是迄今为止最常见的病因（n = 19 例）。
未接受治疗的患者和接受 KTx 前血浆置换预防的患者的累积观察 10 年内复发率分别为 0.81（CI 0.30-1.76）和 3.1（CI 0.64-9.16），而接受 Eculizumab 预防治疗的患者则无复发。Eculizumab 剂量的间隔时间根据经典补体途径活性（目标值<30%）进行调整。根据该策略，目前有 12 名患者每 28 天接受一次 Eculizumab，5 名患者每 24-25 天接受一次，3 名患者每 21 天接受一次。
我们的经验支持对既往有 aHUS 病史且接受 KTx 的患者预防性使用 Eculizumab，特别是当分子生物学明确记录补体失调时。
