Center for HUS Prevention, Control and Management at the Pediatric and Dialysis Unit, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, v. Commenda, 9, 20122, Milan, Italy.
Center for HUS Prevention, Control and Management at the Molecular Biology Laboratory, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
Pediatr Nephrol. 2018 Mar;33(3):457-461. doi: 10.1007/s00467-017-3813-2. Epub 2017 Oct 18.
Atypical hemolytic uremic syndrome (aHUS) is a thrombotic microangiopathy (TMA) characterized by platelet consumption, hemolysis, and organ damage. Eculizumab (ECU), a humanized antibody that blocks complement activity, has been successfully used in aHUS, but the best treatment schedule is not yet clear.
Here, we report our experience with ECU maintenance treatment and the interval between subsequent doses being extended based on global classical complement pathway (CCP) activity aimed at <30% for maintaining aHUS into remission.
We report on 38 patients with aHUS, 13 children, 21 female, with a median age of 25.0 years (range 0.5-60) at disease onset treated with ECU standard schedule for a median of 2.6 months (range 0.4-24.6). Once stable TMA remission was obtained, the interval between ECU doses was extended based on complement function, with a target CCP activity of <30%. With this approach, 22 patients regularly receive ECU infusion every 28 days and 16 every 21. During a median observation period on ECU, an extended interval of 26.9 months (range 0.8-80.9), with a cumulative observation period of 1,208 months, none of the patients relapsed.
Monitoring complement activity allows a safe reduction in the frequency of ECU administration in aHUS while keeping the disease in remission.
非典型溶血性尿毒症综合征（aHUS）是一种以血小板消耗、溶血和器官损伤为特征的血栓性微血管病（TMA）。依库珠单抗（ECU）是一种阻断补体活性的人源化抗体，已成功应用于 aHUS，但最佳治疗方案尚不清楚。
在此，我们报告了我们使用 ECU 维持治疗的经验，并根据旨在将经典补体途径（CCP）活性维持在<30%以保持 aHUS缓解的间隔时间来延长后续剂量的间隔时间。
我们报告了 38 例 aHUS 患者的经验，其中 13 例为儿童，21 例为女性，发病时的中位年龄为 25.0 岁（范围 0.5-60），接受 ECU 标准方案治疗的中位时间为 2.6 个月（范围 0.4-24.6）。一旦获得稳定的 TMA 缓解，根据补体功能延长 ECU 剂量间隔，目标 CCP 活性<30%。采用这种方法，22 例患者定期每 28 天接受一次 ECU 输注，16 例患者每 21 天接受一次。在接受 ECU 中位观察期间，间隔时间延长至 26.9 个月（范围 0.8-80.9），累积观察期为 1208 个月，无一例患者复发。
监测补体活性可在保持疾病缓解的同时，安全地减少 aHUS 中 ECU 的给药频率。
