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Optimal duration of treatment with eculizumab in atypical hemolytic uremic syndrome (aHUS)-a question to be addressed in a scientific way.

作者信息

Ariceta Gema

机构信息

Pediatric Nephrology, Hospital Universitari Vall d' Hebron, Universitat Autonoma de Barcelona, Barcelona, Spain.

出版信息

Pediatr Nephrol. 2019 May;34(5):943-949. doi: 10.1007/s00467-019-4192-7. Epub 2019 Jan 28.

DOI:10.1007/s00467-019-4192-7
PMID:30693384
Abstract
摘要

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Optimal duration of treatment with eculizumab in atypical hemolytic uremic syndrome (aHUS)-a question to be addressed in a scientific way.依库珠单抗治疗非典型溶血性尿毒症综合征(aHUS)的最佳疗程——一个有待以科学方式解决的问题。
Pediatr Nephrol. 2019 May;34(5):943-949. doi: 10.1007/s00467-019-4192-7. Epub 2019 Jan 28.
2
Eculizumab in atypical hemolytic uremic syndrome: strategies toward restrictive use.依库珠单抗治疗非典型溶血尿毒综合征:限制使用策略。
Pediatr Nephrol. 2019 Nov;34(11):2261-2277. doi: 10.1007/s00467-018-4091-3. Epub 2018 Nov 6.
3
Practical issues in using eculizumab for children with atypical haemolytic uraemic syndrome in the acute phase: A review of four patients.在急性期使用依库珠单抗治疗非典型溶血性尿毒症综合征患儿的实际问题:4例患者的回顾
Nephrology (Carlton). 2018 Jun;23(6):539-545. doi: 10.1111/nep.13054.
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Eculizumab Modifies Outcomes in Adults with Atypical Hemolytic Uremic Syndrome with Acute Kidney Injury.依库珠单抗改善急性肾损伤成人非典型溶血尿毒综合征的结局。
Am J Nephrol. 2018;48(3):225-233. doi: 10.1159/000492865. Epub 2018 Sep 11.
5
Eculizumab for atypical hemolytic-uremic syndrome. How long should we maintain it?依库珠单抗治疗非典型溶血性尿毒症综合征。我们应该维持治疗多久?
Nefrologia (Engl Ed). 2019 Jul-Aug;39(4):440-442. doi: 10.1016/j.nefro.2019.01.001. Epub 2019 Feb 21.
6
Outcomes in patients with atypical hemolytic uremic syndrome treated with eculizumab in a long-term observational study.在一项长期观察性研究中,用依库珠单抗治疗非典型溶血尿毒综合征患者的结果。
BMC Nephrol. 2019 Apr 10;20(1):125. doi: 10.1186/s12882-019-1314-1.
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Eculizumab Dosing Regimen in Atypical HUS: Possibilities for Individualized Treatment.依库珠单抗剂量方案在非典型溶血尿毒综合征中的应用:实现个体化治疗的可能。
Clin Pharmacol Ther. 2017 Oct;102(4):671-678. doi: 10.1002/cpt.686. Epub 2017 May 26.
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Three months interval therapy of Eculizumab in a patient with atypical hemolytic uremic syndrome with hybrid CFHR1/CFH gene.依库珠单抗对一名携带CFHR1/CFH杂合基因的非典型溶血尿毒综合征患者进行的三个月间隔治疗
CEN Case Rep. 2019 May;8(2):139-143. doi: 10.1007/s13730-019-00383-7. Epub 2019 Feb 4.
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Efficacy of Eculizumab Therapy for Atypical Hemolytic Uremic Syndrome Recurrence and Antibody-Mediated Rejection Progress After Renal Transplantation With Preformed Donor-Specific Antibodies: Case Report.依库珠单抗治疗伴有预先形成的供者特异性抗体的肾移植后非典型溶血性尿毒症综合征复发及抗体介导的排斥反应进展的疗效:病例报告
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J Med Cases. 2025 Jul 19;16(7):254-258. doi: 10.14740/jmc5129. eCollection 2025 Jul.
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Atypical hemolytic uremic syndrome: diagnosis, management, and discontinuation of therapy.非典型溶血性尿毒症综合征:诊断、管理及治疗的终止
Hematology Am Soc Hematol Educ Program. 2024 Dec 6;2024(1):200-205. doi: 10.1182/hematology.2024000543.
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Treatment discontinuation in adults with atypical hemolytic uremic syndrome (aHUS): a qualitative study of international experts' perspectives with associated cost-consequence analysis.

本文引用的文献

1
Eculizumab in atypical hemolytic uremic syndrome: strategies toward restrictive use.依库珠单抗治疗非典型溶血尿毒综合征:限制使用策略。
Pediatr Nephrol. 2019 Nov;34(11):2261-2277. doi: 10.1007/s00467-018-4091-3. Epub 2018 Nov 6.
2
Anticomplement Treatment in Atypical and Typical Hemolytic Uremic Syndrome.抗补体治疗非典型和典型溶血尿毒综合征。
Semin Hematol. 2018 Jul;55(3):150-158. doi: 10.1053/j.seminhematol.2018.04.009. Epub 2018 Apr 20.
3
Clinical and genetic predictors of atypical hemolytic uremic syndrome phenotype and outcome.
成人非典型溶血性尿毒症综合征(aHUS)停药治疗:国际专家观点的定性研究及相关成本效益分析。
BMC Nephrol. 2024 Nov 14;25(1):411. doi: 10.1186/s12882-024-03770-0.
4
Eculizumab in Shiga toxin-producing Escherichia coli hemolytic uremic syndrome: a systematic review.产志贺毒素大肠埃希菌溶血尿毒综合征应用依库珠单抗治疗:系统评价。
Pediatr Nephrol. 2024 May;39(5):1369-1385. doi: 10.1007/s00467-023-06216-1. Epub 2023 Dec 6.
5
Optimizing the Long-Term Therapy with Eculizumab (ECU) for Atypical Hemolytic Uremic Syndrome (aHUS).优化依库珠单抗(ECU)治疗非典型溶血性尿毒症综合征(aHUS)的长期疗法。
Indian J Pediatr. 2024 Feb;91(2):205. doi: 10.1007/s12098-023-04817-0. Epub 2023 Sep 30.
6
Treatment preference and quality of life impact: ravulizumab vs eculizumab for atypical hemolytic uremic syndrome.治疗偏好和生活质量影响:瑞维鲁单抗与依库珠单抗治疗非典型溶血尿毒症综合征。
J Comp Eff Res. 2023 Sep;12(9):e230036. doi: 10.57264/cer-2023-0036. Epub 2023 Jul 29.
7
Successful Discontinuation of Eculizumab in a Pediatric Patient With Atypical Hemolytic Uremic Syndrome and Underlying Systematic Lupus Erythematosus.成功停用依库珠单抗治疗一名患有非典型溶血性尿毒症综合征及潜在系统性红斑狼疮的儿科患者。
Cureus. 2022 May 18;14(5):e25117. doi: 10.7759/cureus.25117. eCollection 2022 May.
8
Eculizumab for paediatric patients with atypical haemolytic uraemic syndrome: full dataset analysis of post-marketing surveillance in Japan.依库珠单抗治疗小儿非典型溶血尿毒综合征:日本上市后监测的全数据集分析。
Nephrol Dial Transplant. 2023 Feb 13;38(2):414-424. doi: 10.1093/ndt/gfac150.
9
Complement inhibitor eculizumab in thrombotic microangiopathy: Single-center case series.补体抑制剂依库珠单抗治疗血栓性微血管病:单中心病例系列
Clin Case Rep. 2022 Mar 15;10(3):e05573. doi: 10.1002/ccr3.5573. eCollection 2022 Mar.
10
Eculizumab discontinuation in atypical haemolytic uraemic syndrome: TMA recurrence risk and renal outcomes.非典型溶血性尿毒症综合征中依库珠单抗的停药:血栓性微血管病复发风险及肾脏结局
Clin Kidney J. 2021 Jan 24;14(9):2075-2084. doi: 10.1093/ckj/sfab005. eCollection 2021 Sep.
临床和遗传预测因素对非典型溶血尿毒综合征表型和结局的影响。
Kidney Int. 2018 Aug;94(2):408-418. doi: 10.1016/j.kint.2018.02.029. Epub 2018 Jun 19.
4
Controversies of the classification of TMA and the terminology of aHUS.血栓性微血管病(TMA)分类及非典型溶血尿毒综合征(aHUS)术语的争议。
Clin Exp Nephrol. 2018 Aug;22(4):979-980. doi: 10.1007/s10157-017-1524-4. Epub 2017 Dec 27.
5
Safety and effectiveness of restrictive eculizumab treatment in atypical haemolytic uremic syndrome.限制型依库珠单抗治疗非典型溶血性尿毒症综合征的安全性和有效性。
Nephrol Dial Transplant. 2018 Apr 1;33(4):635-645. doi: 10.1093/ndt/gfx196.
6
Complement functional tests for monitoring eculizumab treatment in patients with atypical hemolytic uremic syndrome: an update.补体功能检测在监测非典型溶血尿毒综合征患者依库珠单抗治疗中的作用:更新。
Pediatr Nephrol. 2018 Mar;33(3):457-461. doi: 10.1007/s00467-017-3813-2. Epub 2017 Oct 18.
7
Factor H autoantibody is associated with atypical hemolytic uremic syndrome in children in the United Kingdom and Ireland.因子 H 自身抗体与英国和爱尔兰儿童的非典型溶血性尿毒症综合征有关。
Kidney Int. 2017 Nov;92(5):1261-1271. doi: 10.1016/j.kint.2017.04.028. Epub 2017 Jul 24.
8
Eculizumab Dosing Regimen in Atypical HUS: Possibilities for Individualized Treatment.依库珠单抗剂量方案在非典型溶血尿毒综合征中的应用:实现个体化治疗的可能。
Clin Pharmacol Ther. 2017 Oct;102(4):671-678. doi: 10.1002/cpt.686. Epub 2017 May 26.
9
Haemolytic uraemic syndrome.溶血尿毒综合征。
Lancet. 2017 Aug 12;390(10095):681-696. doi: 10.1016/S0140-6736(17)30062-4. Epub 2017 Feb 25.
10
Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients' health needs?及时获取罕见病药物的伦理要求:能否协调经济激励与患者健康需求?
Orphanet J Rare Dis. 2017 Jan 5;12(1):1. doi: 10.1186/s13023-016-0551-7.