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We prove, for the first time to our knowledge, in a genotype matched CaHUS cohort that the 5-year cumulative estimate of ESKD-free survival improved from 39.5% in a control cohort to 85.5% in the eculizumab-treated cohort (hazard ratio, 4.95; 95% confidence interval [CI], 2.75-8.90; P\u00a0= .000; number needed to treat, 2.17 [95% CI, 1.81-2.73]). The outcome of eculizumab treatment is associated with the underlying genotype. Lower serum creatinine, lower platelet count, lower blood pressure, and younger age at presentation as well as shorter time between presentation and the first dose of eculizumab were associated with estimated glomerular filtration rate &amp;gt;60 ml\/min at 6 months in multivariate analysis. The rate of meningococcal infection in the treated cohort was 550 times greater than the background rate in the general population. The relapse rate upon eculizumab withdrawal was 1 per 9.5 person years for patients with a pathogenic mutation and 1 per 10.8 person years for those with a variant of uncertain significance. No relapses were recorded in 67.3 person years off eculizumab in those with no rare genetic variants. Eculizumab was restarted in 6 individuals with functioning kidneys in whom it had been stopped, with no individual progressing to ESKD. We demonstrated that biallelic pathogenic mutations in RNA-processing genes, including EXOSC3, encoding an essential part of the RNA exosome, cause eculizumab nonresponsive aHUS. Recessive HSD11B2 mutations causing apparent mineralocorticoid excess may also present with thrombotic microangiopathy.<\/jats:p>","DOI":"10.1182\/blood.2022018833","type":"journal-article","created":{"date-parts":[[2023,6,27]],"date-time":"2023-06-27T16:04:01Z","timestamp":1687881841000},"page":"1371-1386","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":83,"title":["Atypical hemolytic uremic syndrome in the era of terminal complement inhibition: an observational cohort study"],"prefix":"10.1182","volume":"142","author":[{"given":"Vicky","family":"Brocklebank","sequence":"first","affiliation":[{"name":"1National Renal Complement Therapeutics Centre, Newcastle upon Tyne Hospitals National Health Service Foundation Trust, Newcastle upon Tyne, United Kingdom"},{"name":"2Complement Therapeutics Research Group, Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9852-0095","authenticated-orcid":false,"given":"Patrick R.","family":"Walsh","sequence":"additional","affiliation":[{"name":"1National Renal Complement Therapeutics Centre, Newcastle upon Tyne Hospitals National Health Service Foundation Trust, Newcastle upon Tyne, United Kingdom"},{"name":"2Complement Therapeutics Research Group, Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kate","family":"Smith-Jackson","sequence":"additional","affiliation":[{"name":"1National Renal Complement Therapeutics Centre, Newcastle upon Tyne Hospitals National Health Service Foundation Trust, Newcastle upon Tyne, United Kingdom"},{"name":"2Complement Therapeutics Research Group, Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4632-9640","authenticated-orcid":false,"given":"Thomas M.","family":"Hallam","sequence":"additional","affiliation":[{"name":"2Complement Therapeutics Research Group, Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1312-5411","authenticated-orcid":false,"given":"Kevin J.","family":"Marchbank","sequence":"additional","affiliation":[{"name":"1National Renal Complement Therapeutics Centre, Newcastle upon Tyne Hospitals National Health Service Foundation Trust, Newcastle upon Tyne, United Kingdom"},{"name":"2Complement Therapeutics Research Group, Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Valerie","family":"Wilson","sequence":"additional","affiliation":[{"name":"1National Renal Complement Therapeutics Centre, Newcastle upon Tyne Hospitals National Health Service Foundation Trust, Newcastle upon Tyne, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2387-4918","authenticated-orcid":false,"given":"Theophile","family":"Bigirumurame","sequence":"additional","affiliation":[{"name":"3Population Health Sciences Institute, Newcastle University, Newcastle upon Tyne, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3049-2395","authenticated-orcid":false,"given":"Tina","family":"Dutt","sequence":"additional","affiliation":[{"name":"4Department of Haematology, Royal Liverpool University Hospital, Liverpool, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4051-2620","authenticated-orcid":false,"given":"Emma K.","family":"Montgomery","sequence":"additional","affiliation":[{"name":"1National Renal Complement Therapeutics Centre, Newcastle upon Tyne Hospitals National Health Service Foundation Trust, Newcastle upon Tyne, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9434-1521","authenticated-orcid":false,"given":"Michal","family":"Malina","sequence":"additional","affiliation":[{"name":"1National Renal Complement Therapeutics Centre, Newcastle upon Tyne Hospitals National Health Service Foundation Trust, Newcastle upon Tyne, United Kingdom"},{"name":"2Complement Therapeutics Research Group, Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom"},{"name":"5Great North Children's Hospital, Sir James Spence Institute, Royal Victoria Infirmary, Newcastle, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Edwin K. 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