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Lookup NU author(s): Professor Tim Goodship
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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.
Author(s): Cullinan N, Gorman KM, Riordan M, Waldron M, Goodship THJ, Awan A
Publication type: Article
Publication status: Published
Journal: Pediatrics
Year: 2015
Volume: 135
Issue: 6
Pages: E1506-E1509
Print publication date: 01/06/2015
Acceptance date: 25/02/2015
ISSN (print): 0031-4005
ISSN (electronic): 1098-4275
Publisher: American Academy of Pediatrics
URL: http://dx.doi.org/10.1542/peds.2014-3503
DOI: 10.1542/peds.2014-3503
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