治疗严重肾血栓性微血管病的非浆膜腔穿刺方法:7 个病例的报告。

IF 9.4 1区 医学 Q1 UROLOGY & NEPHROLOGY American Journal of Kidney Diseases Pub Date : 2024-09-27 DOI:10.1053/j.ajkd.2024.06.022
Marie Frimat, Mehdi Maanaoui, Nora Schwotzer, Celine Lebas, Arnaud Lionet, Sylvain Dubucquoi, Viviane Gnemmi, Fadi Fakhouri, François Provôt
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引用次数: 0

摘要

C5 受体阻滞剂是治疗补体介导的溶血性尿毒症综合征(CM-HUS)的公认疗法。然而,CM-HUS 缺乏明确的检测方法,因此,尽管该适应症存在并发症且证据有限,但在进一步评估之前,血浆置换仍是常用的一线疗法。最近的专家意见支持将 C5 受体阻滞剂作为治疗严重肾血栓性微血管病(TMA)的初始疗法。本项回顾性单中心研究报告了采用无浆膜腔穿刺方法治疗的七名患者。所有患者都出现了严重的肾脏 TMA 症状和较低的 French 评分,并在入院后中位 9 小时内及时接受了 900 毫克的依库珠单抗治疗。血液学恢复迅速,六名患者的肾功能在 6.5 天内得到改善,中位住院时间为 16 天。没有使用血浆置换术进行抢救。我们报告了七例怀疑为 CM-HUS 的严重肾脏 TMA 患者的早期 C5 受体阻滞剂和免血浆置换策略,显示了良好的初步效果。这种方法的有效性和安全性还需要临床试验来证实。解决C5受体阻滞疗法的高昂费用问题并探索具有成本效益的替代疗法对于成人肾脏TMA靶向疗法的更广泛实施和普及也至关重要。
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Non-plasmapheresis Approaches for Managing Severe Renal Thrombotic Microangiopathy: A Report of 7 Cases.

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.

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来源期刊
American Journal of Kidney Diseases
American Journal of Kidney Diseases 医学-泌尿学与肾脏学
CiteScore
20.40
自引率
2.30%
发文量
732
审稿时长
3-8 weeks
期刊介绍: The American Journal of Kidney Diseases (AJKD), the National Kidney Foundation's official journal, is globally recognized for its leadership in clinical nephrology content. Monthly, AJKD publishes original investigations on kidney diseases, hypertension, dialysis therapies, and kidney transplantation. Rigorous peer-review, statistical scrutiny, and a structured format characterize the publication process. Each issue includes case reports unveiling new diseases and potential therapeutic strategies.
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