{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,10]],"date-time":"2026-06-10T09:46:32Z","timestamp":1781084792874,"version":"3.54.1"},"reference-count":16,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2023,11,20]],"date-time":"2023-11-20T00:00:00Z","timestamp":1700438400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100005567","name":"H. Lee Moffitt Cancer Center and Research Institute","doi-asserted-by":"publisher","award":["P30CA076292\u201016"],"award-info":[{"award-number":["P30CA076292\u201016"]}],"id":[{"id":"10.13039\/100005567","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004361","name":"Krebsliga Schweiz","doi-asserted-by":"publisher","award":["KLS\u20103352\u201002\u20102014"],"award-info":[{"award-number":["KLS\u20103352\u201002\u20102014"]}],"id":[{"id":"10.13039\/501100004361","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["European J of Haematology"],"published-print":{"date-parts":[[2024,3]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Complement\u2010mediated thrombotic microangiopathy (CM\u2010TMA), also called atypical hemolytic uremic syndrome (aHUS), is a difficult\u2010to\u2010diagnose rare disease that carries severe morbidity and mortality. Anti\u2010C5 monoclonal antibodies (aC5\u2010mab) are standard treatments, but large studies and long\u2010term data are scarce. Here, we report our single institution experience to augment the knowledge of CM\u2010TMA treated with aC5\u2010mab therapy.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>We aimed to assess the short and long\u2010term effects of aC5\u2010mab in patients diagnosed with CM\u2010TMA treated outside of a clinical trial. This was a retrospective study. We included all patients diagnosed with CM\u2010TMA and treated with aC5\u2010mab at our institution. There were no exclusion criteria. Endpoints included complete TMA response (CR) defined as normalization of hematological parameters and \u226525% improvement in serum creatinine (Cr) from baseline in patients with renal disease, relapse defined as losing the previously achieved CR, morbidity, adverse events, and survival.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>We found 28 patients with CM\u2010TMA treated with aC5\u2010mab. The median age was 50\u2009years. Baseline laboratories: platelet counts 93\u2009\u00d7\u200910<jats:sup>9<\/jats:sup>\/L, hemoglobin 8.6\u2009g\/dL, lactate dehydrogenase 1326\u2009U\/L, serum Cr 4.7\u2009mg\/dL, and estimated glomerular filtration rate 19\u2009mL\/min. One individual was on renal replacement therapy (RRT) and 10 initiated RRT within 5\u2009days of the first dose of aC5\u2010mab. Genetic variants associated with CM\u2010TMA included mutations in C3, CFB, CFH, CFHR1\/3, CFI, and MCP. The mean duration of hospitalization was 24\u2009days. The median time to initiation of aC5\u2010mab was 10\u2009days. Sixteen subjects received RRT. At the time of hospital discharge, 27 were alive, 14 remained on RRT, and 4 had a CR. At 6\u2009months, 23 patients were alive, 18 continued aC5\u2010mab, 8 remained on RRT, and 9 had a CR. At the last follow\u2010up visit past 6\u2009months, 20 were alive, 14 continued aC5\u2010mab, 5 remained on RRT, 12 had a CR, and 1 was lost to follow\u2010up.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Our study provides real\u2010world experience and insight into the long\u2010term outcomes of CM\u2010TMA treated with aC5\u2010mab. Our findings validate that CM\u2010TMA is an aggressive disease with significant morbidity and mortality, and confirm that aC5\u2010mab is a relatively effective therapy for CM\u2010TMA. Our study adds practical, real\u2010world experience to the literature, but future research remains imperative.<\/jats:p><\/jats:sec>","DOI":"10.1111\/ejh.14136","type":"journal-article","created":{"date-parts":[[2023,11,21]],"date-time":"2023-11-21T22:09:15Z","timestamp":1700604555000},"page":"450-457","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Complement\u2010mediated thrombotic microangiopathy treated with anticomplement protein 5 therapy, a retrospective study"],"prefix":"10.1111","volume":"112","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-1041-4429","authenticated-orcid":false,"given":"Damian A.","family":"Laber","sequence":"first","affiliation":[{"name":"Department of Satellite and Community Oncology H. Lee Moffitt Cancer Center and Research Institute  Tampa Florida USA"},{"name":"Department of Oncologic Sciences, Morsani College of Medicine University of South Florida  Tampa Florida USA"},{"name":"Department of Internal Medicine, Morsani College of Medicine University of South Florida  Tampa Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8528-1029","authenticated-orcid":false,"given":"Parth C.","family":"Patel","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine, Morsani College of Medicine University of South Florida  Tampa Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Constantine N.","family":"Logothetis","sequence":"additional","affiliation":[{"name":"Divisions of Hematology and Oncology Washington University School of Medicine  St. Louis Missouri USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ankita K.","family":"Patel","sequence":"additional","affiliation":[{"name":"Department of Satellite and Community Oncology H. Lee Moffitt Cancer Center and Research Institute  Tampa Florida USA"},{"name":"Department of Oncologic Sciences, Morsani College of Medicine University of South Florida  Tampa Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4305-2535","authenticated-orcid":false,"given":"Michael","family":"Jaglal","sequence":"additional","affiliation":[{"name":"Department of Satellite and Community Oncology H. Lee Moffitt Cancer Center and Research Institute  Tampa Florida USA"},{"name":"Department of Oncologic Sciences, Morsani College of Medicine University of South Florida  Tampa Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mintallah","family":"Haider","sequence":"additional","affiliation":[{"name":"Department of Satellite and Community Oncology H. 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