Journal
QJM-AN INTERNATIONAL JOURNAL OF MEDICINE
Volume 109, Issue 1, Pages 27-33Publisher
OXFORD UNIV PRESS
DOI: 10.1093/qjmed/hcv082
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- Alexion Pharmaceuticals
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Results: In the first year of service, 43 aHUS patients received eculizumab, 15 children and 28 adults. Twenty-three were new patients and 20 prevalent. Fifteen of the 23 new patients required dialysis before eculizumab was started, 8 of these recovered renal function. Twelve of the 20 prevalent patients who received eculizumab were transplant patients, 8 with prophylactic use and 4 for recurrent disease; the outcome in all was good. Eculizumab was withdrawn in 14 patients, 5 were patients who had not recovered renal function. In 3 of the 14 patients, it was necessary to reintroduce eculizumab because of recurrent disease (2 extra-renal and 1 renal). There were 2 deaths in the 43 patients, and neither was associated with use of eculizumab. There were no episodes of meningococcal disease. Conclusions: The establishment of this national service has enabled aHUS patients in England to receive eculizumab when they need it for as long as they need it.
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