Real-World Effectiveness of Ravulizumab Among C5 Inhibitor-Naive Patients With Atypical Hemolytic Uremic Syndrome: A Physician Panel-Based Chart Review (aHUS IMPACT Study).

Rationale & Objective: Atypical hemolytic uremic syndrome (aHUS) is a rare form of thrombotic microangiopathy (TMA) caused by complement dysregulation. Ravulizumab, a complement C5 inhibitor (C5i), is approved for aHUS; however, published evidence in a real-world setting is limited. Study Design: Retrospective, longitudinal, physician panel-based chart review. Setting & Population: C5i-naive adults with aHUS in the United States treated with ravulizumab. Physicians randomly selected 1-5 patients who had ≥6 months of follow-up after ravulizumab initiation; patients who died within 6 months of initiation were eligible. Exposures or Predictors: Ravulizumab. Outcomes: The clinical outcomes evaluated included hematologic and renal outcomes, complete TMA response (a composite hematolgic/renal endpoint), and dialysis use. Analytical Approach: Descriptive statistics, Kaplan-Meier estimators, and generalized linear models. Results: Overall, 79 C5i-naive adults with aHUS (enrolled by 31 physicians) initiated ravulizumab and were included in the study. Statistically significant improvements from baseline occurred as early as day 4 (lactate dehydrogenase and percent change in serum creatinine; both Limitations: The study design relies on available medical record data and has potential responder bias. Conclusions: This study supports the immediate and sustained benefits of initiating ravulizumab in patients with aHUS as seen by the early response and continued improvement in clinical outcomes.