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Topics:
Pediatric Hematology/Oncology • Internal Medicine • Hematology • Pediatric Hematology • Benign Hematology
In which patients with atypical HUS would you consider eculizumab discontinuation?
Given the high cost of eculizumab, are there patient characteristics that inform which patients, if any, should be on indefinite therapy versus a time-limited course?
Answer from: at Community Practice
My approach to eculizumab cessation in aHUS is to at least consider cessation in all patients not only given the high cost of the drug, but also given the risk of meningococcemia, which is incompletely protected against by vaccines.In treating aHUS, I initiate eculizumab (and preventive therapy for ...
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Answer from: Medical Oncologist at Community Practice
I agree with all the points made by Dr. @Marshall A. Mazepa, but would add that genetic information can be useful in informing the discussion. Fakhouri et al recently published prospective data of 55 patients who discontinued eculizumab, 51% of which had complement gene rare variants. Overall, 23% e...