{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,10]],"date-time":"2026-10-10T06:11:15Z","timestamp":1791612675728,"version":"4.3.4"},"reference-count":17,"publisher":"American Academy of Pediatrics (AAP)","issue":"6","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2015,6,1]]},"abstract":"<jats:p>Atypical hemolytic uremic syndrome (aHUS) is caused by dysregulation of the complement system, leading to complement overactivation. A humanized anti-C5 monoclonal antibody, eculizumab, has been available for the treatment of aHUS since 2011. The long-term safety and efficacy of this novel drug in the pediatric population remain under review. We present a child with a hybrid CFH\/CFHR3 gene who, having had multiple disease relapses despite optimal treatment with plasma exchange, commenced eculizumab therapy in August 2010. She remains relapse free in follow-up at 52 months, and treatment has been well tolerated. The risk of meningococcal disease during this treatment is recognized. Despite vaccination against meningococcal disease and appropriate antibiotic prophylaxis, our patient developed meningococcal bacteremia 30 months into treatment. She presented with nonspecific symptoms but recovered without sequelae with appropriate treatment. We recommend that children be vaccinated against invasive meningococcal infection before beginning eculizumab therapy and take appropriate antibiotic prophylaxis during treatment, and we suggest that vaccine responses should be checked and followed annually. Clinicians need to maintain a high index of suspicion for invasive meningococcal disease. Neither vaccination nor antibiotic prophylaxis provides complete protection in patients on eculizumab therapy. The appropriate dosage of eculizumab needed to achieve remission in aHUS in the pediatric population is unknown. Having achieved remission in our patient, we monitor eculizumab and CH50 levels to evaluate ongoing blockade of the terminal complement cascade. Such information may help guide dosing intervals in the future.<\/jats:p>","DOI":"10.1542\/peds.2014-3503","type":"journal-article","created":{"date-parts":[[2015,5,5]],"date-time":"2015-05-05T00:11:19Z","timestamp":1430784679000},"page":"e1506-e1509","source":"Crossref","is-referenced-by-count":42,"title":["Case Report: Benefits and Challenges of Long-term Eculizumab in Atypical Hemolytic Uremic Syndrome"],"prefix":"10.1542","volume":"135","author":[{"given":"Noelle","family":"Cullinan","sequence":"first","affiliation":[{"name":"The Department for Paediatric Nephrology & Transplantation, The Children\u2019s University Hospital, Dublin 1, Ireland; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathleen Mary","family":"Gorman","sequence":"additional","affiliation":[{"name":"The Department for Paediatric Nephrology & Transplantation, The Children\u2019s University Hospital, Dublin 1, Ireland; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Riordan","sequence":"additional","affiliation":[{"name":"The Department for Paediatric Nephrology & Transplantation, The Children\u2019s University Hospital, Dublin 1, Ireland; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mary","family":"Waldron","sequence":"additional","affiliation":[{"name":"The Department for Paediatric Nephrology & Transplantation, The Children\u2019s University Hospital, Dublin 1, Ireland; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Timothy H.J","family":"Goodship","sequence":"additional","affiliation":[{"name":"Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Atif","family":"Awan","sequence":"additional","affiliation":[{"name":"The Department for Paediatric Nephrology & Transplantation, The Children\u2019s University Hospital, Dublin 1, Ireland; and"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"417","published-online":{"date-parts":[[2015,6,1]]},"reference":[{"issue":"10","key":"2021121110162192100_B1","doi-asserted-by":"crossref","first-page":"1967","DOI":"10.1007\/s00467-014-2817-4","article-title":"An audit analysis of a guideline for the investigation and initial therapy of diarrhea negative (atypical) hemolytic uremic syndrome.","volume":"29","author":"Johnson","year":"2014","journal-title":"Pediatr Nephrol"},{"issue":"4","key":"2021121110162192100_B2","doi-asserted-by":"crossref","first-page":"554","DOI":"10.2215\/CJN.04760512","article-title":"Genetics and outcome of atypical hemolytic uremic syndrome: a nationwide French series comparing children and adults.","volume":"8","author":"Fremeaux-Bacchi","year":"2013","journal-title":"Clin J Am Soc Nephrol"},{"issue":"10","key":"2021121110162192100_B3","doi-asserted-by":"crossref","first-page":"1844","DOI":"10.2215\/CJN.02210310","article-title":"Relative role of genetic complement abnormalities in sporadic and familial aHUS and their impact on clinical phenotype.","volume":"5","author":"Noris","year":"2010","journal-title":"Clin J Am Soc Nephrol"},{"key":"2021121110162192100_B4","doi-asserted-by":"crossref","first-page":"60","DOI":"10.1186\/1750-1172-6-60","article-title":"Atypical hemolytic uremic syndrome.","volume":"6","author":"Loirat","year":"2011","journal-title":"Orphanet J Rare Dis"},{"issue":"6","key":"2021121110162192100_B5","doi-asserted-by":"crossref","first-page":"508","DOI":"10.1016\/j.semnephrol.2013.08.003","article-title":"Atypical hemolytic uremic syndrome.","volume":"33","author":"Kavanagh","year":"2013","journal-title":"Semin Nephrol"},{"issue":"2","key":"2021121110162192100_B6","doi-asserted-by":"crossref","first-page":"591","DOI":"10.1182\/blood-2011-03-339903","article-title":"A novel hybrid CFH\/CFHR3 gene generated by a microhomology-mediated deletion in familial atypical hemolytic uremic syndrome.","volume":"119","author":"Francis","year":"2012","journal-title":"Blood"},{"issue":"4","key":"2021121110162192100_B7","doi-asserted-by":"crossref","first-page":"687","DOI":"10.1007\/s00467-008-0964-1","article-title":"Guideline for the investigation and initial therapy of diarrhea-negative hemolytic uremic syndrome.","volume":"24","author":"Ariceta","year":"2009","journal-title":"Pediatr Nephrol"},{"issue":"3","key":"2021121110162192100_B8","doi-asserted-by":"crossref","first-page":"199","DOI":"10.1016\/j.molimm.2013.05.224","article-title":"Complement therapy in atypical haemolytic uraemic syndrome (aHUS).","volume":"56","author":"Wong","year":"2013","journal-title":"Mol Immunol"},{"issue":"5","key":"2021121110162192100_B9","doi-asserted-by":"crossref","first-page":"542","DOI":"10.1056\/NEJMc0808527","article-title":"Eculizumab for atypical hemolytic-uremic syndrome [published correction appears in N Engl J Med. 2009;360(23):2487].","volume":"360","author":"N\u00fcrnberger","year":"2009","journal-title":"N Engl J Med"},{"issue":"25","key":"2021121110162192100_B10","doi-asserted-by":"crossref","first-page":"6786","DOI":"10.1182\/blood-2011-02-333997","article-title":"Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival.","volume":"117","author":"Kelly","year":"2011","journal-title":"Blood"},{"issue":"11","key":"2021121110162192100_B11","doi-asserted-by":"crossref","first-page":"2085","DOI":"10.1007\/s00467-011-1989-4","article-title":"Eculizumab in atypical hemolytic uremic syndrome: long-term clinical course and histological findings.","volume":"26","author":"Tschumi","year":"2011","journal-title":"Pediatr Nephrol"},{"issue":"11","key":"2021121110162192100_B12","doi-asserted-by":"crossref","first-page":"643","DOI":"10.1038\/nrneph.2012.214","article-title":"Use of eculizumab for atypical hemolytic uremic syndrome and C3 glomerulopathies.","volume":"8","author":"Zuber","year":"2012","journal-title":"Nat Rev Nephrol"},{"issue":"4","key":"2021121110162192100_B13","doi-asserted-by":"crossref","first-page":"621","DOI":"10.1007\/s00467-010-1719-3","article-title":"Efficacy of eculizumab in a patient with factor-H-associated atypical hemolytic uremic syndrome.","volume":"26","author":"Lapeyraque","year":"2011","journal-title":"Pediatr Nephrol"},{"issue":"12","key":"2021121110162192100_B14","doi-asserted-by":"crossref","first-page":"2323","DOI":"10.1007\/s00467-012-2276-8","article-title":"Long-term eculizumab improves clinical outcomes in atypical hemolytic uremic syndrome.","volume":"27","author":"Vilalta","year":"2012","journal-title":"Pediatr Nephrol"},{"issue":"23","key":"2021121110162192100_B15","doi-asserted-by":"crossref","first-page":"2169","DOI":"10.1056\/NEJMoa1208981","article-title":"Terminal complement inhibitor eculizumab in atypical hemolytic-uremic syndrome.","volume":"368","author":"Legendre","year":"2013","journal-title":"N Engl J Med"},{"issue":"11","key":"2021121110162192100_B16","doi-asserted-by":"crossref","first-page":"e003573","DOI":"10.1136\/bmjopen-2013-003573","article-title":"A systematic review of eculizumab for atypical haemolytic uraemic syndrome (aHUS).","volume":"3","author":"Rathbone","year":"2013","journal-title":"BMJ Open"},{"issue":"3","key":"2021121110162192100_B17","doi-asserted-by":"crossref","first-page":"819","DOI":"10.1111\/ajt.12032","article-title":"Meningococcal sepsis complicating eculizumab treatment despite prior vaccination.","volume":"13","author":"Struijk","year":"2013","journal-title":"Am J Transplant"}],"container-title":["Pediatrics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/publications.aap.org\/pediatrics\/article-pdf\/135\/6\/e1506\/913127\/peds_2014-3503.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"syndication"},{"URL":"https:\/\/publications.aap.org\/pediatrics\/article-pdf\/135\/6\/e1506\/913127\/peds_2014-3503.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2021,12,11]],"date-time":"2021-12-11T10:16:37Z","timestamp":1639217797000},"score":1,"resource":{"primary":{"URL":"https:\/\/publications.aap.org\/pediatrics\/article\/135\/6\/e1506\/75840\/Case-Report-Benefits-and-Challenges-of-Long-term"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2015,6,1]]},"references-count":17,"journal-issue":{"issue":"6","published-online":{"date-parts":[[2015,6,1]]},"published-print":{"date-parts":[[2015,6,1]]}},"URL":"https:\/\/doi.org\/10.1542\/peds.2014-3503","relation":{},"ISSN":["0031-4005","1098-4275"],"issn-type":[{"value":"0031-4005","type":"print"},{"value":"1098-4275","type":"electronic"}],"subject":[],"published":{"date-parts":[[2015,6,1]]}}}