Abstract
• At the 3-year update: – The median treatment duration was 156 weeks, with a range of 26 to 176 weeks. – Sixteen patients completed ≥30 months of treatment. – Ten patients remained enrolled in the trial at data cutoff. • Patients exited the study following marketing authorization of eculizumab and as commercial supply became available in the study countries. • All patients will be enrolled in an observational 5-year long-term follow-up study (US National Institutes of Health ClinicalTrials.gov Identifier: NCT01522170).
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