Eculizumab (ECU) Inhibits Thrombotic Microangiopathy (TMA) and Improves Renal Function in Adult Atypical Hemolytic Uremic Syndrome (aHUS) Patients (Pts)
Eculizumab (ECU) Inhibits Thrombotic Microangiopathy (TMA) and Improves Renal Function in Adult Atypical Hemolytic Uremic Syndrome (aHUS) Patients (Pts)
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依库珠单抗 (ECU) 抑制成人非典型溶血性尿毒症综合征 (aHUS) 患者的血栓性微血管病 (TMA) 并改善肾功能（Pts）
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发表时间：
2013
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通讯作者：
C. Legendre
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作者：
F. Fakhouri;M. Hourmant;J. Campistol;S. Cataland;M. Espinosa;O. Gaber;J. Menne;E. Minetti;F. Provôt;E. Rondeau;P. Ruggenenti;L. Weekers;M. Ogawa;C. Bedrosian;C. Legendre
[FR-OR 057]依库珠单抗（ECU）抑制血栓性微血管病（TMA）并改善成人非典型溶血性尿毒症综合征（阿胡斯）患者的肾功能（Pts）Fadi Fakhouri，MD，PhD，Maryvonne Hoursman，MD，PhD，Josep M.斯佩罗？坎皮斯托尔Cataland，MD，Mario埃斯皮诺萨，MD，A. Osama Gaber，MD，Jan Menne，MD，Enrico E. Minetti，MD，Francois Provot，MD，Eric Rondeau，MD，PhD，Piero Ruggenenti，Laurent E. Weekers，MD，Masayo Ogawa，MD，Camille L. Bedrosian，MD，Christophe M. Legendre，MD. C10-004研究组。6：18 PM 6：30 PM背景：阿胡斯是一种罕见的、严重的、遗传性的、危及生命的慢性补体介导的TMA疾病。ECU是一种末端补体抑制剂，已被批准用于治疗阿胡斯。在这里，我们报告了在阿胡斯中进行的最大前瞻性研究中ECU在成人患者中的安全性和有效性结果。方法：这是一项在筛选时阿胡斯和血小板<LLN的成人患者（≥18岁）中进行的ECU单臂、II期试验。入选时无需既往血浆置换或输注（PE/PI）。主要终点是26 wks. Results完全TMA反应的患者比例：41例患者入组，38例（93%）接受了26周的治疗。30例患者（73%）为新诊断（至治疗开始的中位持续时间为2周）。6例患者在当前临床表现期间无PE/PI。在第26周，30例患者（73%）达到主要终点。24/41例患者（59%）在BL时接受透析，其中20例患者在第26周前停药。平均（95% CI）eGFR较基线增加26.1 mL/min/1.73 m（19.8; 32.4：P<0.0001）。两名患者在基线时未接受透析，在治疗期间开始透析，并在26周内保持透析。QoL显著改善。ECU通常是安全的，耐受性良好。2例患者发生脑膜炎球菌感染; 1例患者继续ECU。无患者死亡。结论：ECU使血液学参数正常化，显著改善肾功能和生活质量。基线时83%的透析患者能够在第26周停止治疗。该前瞻性研究的结果证实，ECU抑制成人阿胡斯患者中补体介导的TMA。研究正在进行中。资金来源：商业SupportAlexion制药课程：年度会议：摘要会议会话：肾小球疾病：新的方法来预测和治疗日期/时间：星期五，2013年11月8日4：30下午6：30下午位置：房间206个人作者披露可在肾脏周2013披露摘要，这是提供给每个会议参与者或要求在11月。
[FR-OR057] Eculizumab (ECU) Inhibits Thrombotic Microangiopathy (TMA) and Improves Renal Function in Adult Atypical Hemolytic Uremic Syndrome (aHUS) Patients (Pts) Fadi Fakhouri, MD, PhD, Maryvonne Hourmant, MD, PhD, Josep M. Campistol, Spero R. Cataland, MD, Mario Espinosa, MD, A. Osama Gaber, MD, Jan Menne, MD, Enrico E. Minetti, MD, Francois Provot, MD, Eric Rondeau, MD, PhD, Piero Ruggenenti, Laurent E. Weekers, MD, Masayo Ogawa, MD, Camille L. Bedrosian, MD, Christophe M. Legendre, MD. The C10-004 Study Group. 6:18 PM 6:30 PM Background: aHUS is a rare, severe, genetic, life-threatening disease of chronic complement-mediated TMA. ECU, a terminal complement inhibitor, is approved for the treatment of aHUS. Here, we report safety and efficacy results of ECU in adult pts from the largest prospective study performed in aHUS. Methods: This was a single-arm, Phase 2 trial of ECU in adult pts (≥18 yrs) with aHUS and platelets <LLN at screening. Prior plasma exchange or infusion (PE/PI) was not required for inclusion. The primary endpoint was the proportion of pts with complete TMA response at 26 wks. Results: 41 pts enrolled and 38 (93%) received 26 wks of treatment. 30 pts (73%) were newly diagnosed (median duration to treatment initiation of 2 weeks). Six pts had no PE/PI during the current clinical manifestation. At wk 26, 30 pts (73%) achieved the primary endpoint. 24/41 pts (59%) were on dialysis at BL, 20 of whom discontinued by wk 26. Mean (95% CI) eGFR increase from baseline was 26.1 mL/min/1.73 m (19.8; 32.4: P<0.0001). Two pts who were not on dialysis at baseline initiated during the treatment period and remained on dialysis through 26 wks. QoL significantly improved. ECU was generally safe and well tolerated. Two pts had meningococcal infections; one pt continued ECU. No pts died. Conclusions: ECU normalized hematologic parameters and significantly improved renal function and QoL. 83% of pts on dialysis at baseline were able to discontinue by wk 26. The results of this prospective study confirm that ECU inhibits complement-mediated TMA in adult aHUS pts. The study is ongoing. Funding: Commercial SupportAlexion Pharmaceuticals Course: Annual Meeting: Abstract Sessions Session: Glomerular Diseases: New Approaches to Prognosis and Treatment Date/Time: Friday, November 8, 2013 4:30 PM 6:30 PM Location: Room 206 Individual author disclosures are available in the Kidney Week 2013 Disclosure Digest which is available to each meeting participant or upon request in November.