Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment
Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment
This was a prospective, open-label study with no participant randomization. Treatment for
 aHUS was observational and at the discretion of the treating physician. The purpose of this
 study was to assess disease manifestations of complement-mediated thrombotic microangiopathy
 (TMA) and evaluate potential clinical predictors of disease manifestations and progression in
 participants with aHUS with or without eculizumab treatment in the clinical setting.
Inclusion Criteria:

 1. Currently receiving eculizumab treatment in the M11-001 aHUS Registry

 2. Two normal platelet counts at least 4 weeks apart

 3. Two normal lactate dehydrogenase levels at least 4 weeks apart

 4. Willing, committed, and able to return for all clinic visits and complete all
 study-related procedures

 5. Participant or participant's parent/legal guardian must have been willing and able to
 give written informed consent. Participant (if minor) must have been willing to give
 written informed assent (if applicable as determined by the central Institutional
 Review Boards/Independent Ethics Committees)

 Exclusion Criteria:

 1. Any prior eculizumab treatment discontinuation

 2. On chronic dialysis (defined as ≥3 months on dialysis)

 3. Currently participating in another complement inhibitor trial

 4. Life expectancy of <6 months

 5. Participant or participant's parent/legal guardian was unable to give written informed
 consent. Participant (if minor) was unable to give written informed assent (if
 applicable as determined by the central Institutional Review Boards/Independent Ethics
 Committees)