C5-blockers are the established treatment for complement-mediated hemolytic uremic syndrome (CM-HUS). However, CM-HUS, lacking a definitive test, prompts plasma exchanges as a common first-line therapy, pending further assessments, despite complications and limited evidence in this indication. Recent experts’ opinion endorses C5-blockers as the initial treatment for severe renal thrombotic microangiopathy (TMA). This retrospective, single-center study reports a series of 7 patients treated with a plasmapheresis-free approach. All patients presented with severe renal TMA symptoms and low French score and received prompt 900mg eculizumab within a median of 9 hours from admission. Hematological recovery was rapid, and renal function improved in 6 patients within 6.5 days, with a median hospital stay of 16 days. No rescue plasmapheresis was used. We report 7 cases of an early C5-blocker and plasmapheresis-free strategy in severe renal TMA suspicious for CM-HUS, demonstrating promising initial results. Clinical trials are needed to confirm the efficacy and safety of this approach. Addressing the high cost of C5-blocking therapies and exploring cost-effective alternatives is also crucial for broader implementation and accessibility in targeted therapies for adult renal TMA.

中文翻译：

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治疗严重肾血栓性微血管病的非血浆置换方法：7 例病例报告


C5 受体阻滞剂是补体介导的溶血尿毒症综合征 （CM-HUS） 的既定治疗方法。然而，CM-HUS 缺乏确定性检查，促使血浆置换作为常见的一线治疗，尽管存在并发症且该适应证的证据有限，但仍有待进一步评估。最近的专家意见支持将 C5 受体阻滞剂作为严重肾血栓性微血管病 （TMA） 的初始治疗方法。这项回顾性单中心研究报告了 7 例接受无血浆置换方法治疗的患者。所有患者均出现严重的肾脏 TMA 症状和低 French 评分，并在入院后中位 9 小时内及时接受 900 毫克依库珠单抗治疗。血液学恢复迅速，6 例患者在 6.5 天内肾功能改善，中位住院时间为 16 天。未使用挽救性血浆置换。我们报道了 7 例在疑似 CM-HUS 的严重肾 TMA 中的早期 C5 阻滞剂和无血浆置换策略，显示出有希望的初步结果。需要临床试验来确认这种方法的有效性和安全性。解决 C5 阻断疗法的高成本问题并探索具有成本效益的替代方案对于成人肾脏 TMA 靶向治疗的更广泛实施和可及性也至关重要。