Kidney Week

Abstract: FR-PO0833

Patients with Atypical Hemolytic Uremic Syndrome and Intensive Care Unit Support: Analysis of a Ravulizumab Phase 3 Study

Session Information

• Glomerular Diseases: Practice and New Concepts Shaping Modern Care
  October 23, 2026 | Location: Exhibit Hall A, Convention Center
  Abstract Time: 10:00 AM - 12:00 PM

Category: Glomerular Diseases

• 1402 Glomerular Diseases: Clinical, Outcomes, and Therapeutics

Authors

• Azoulay, Elie, Medical Intensive Care Unit Department, AP-HP, Saint-Louis University Hospital, Paris-Cité University, Paris, France

Elie Azoulay, MD, PhD

• Abbasi, Saad, Alexion, AstraZeneca Rare Disease, Boston, Massachusetts, United States

Saad Abbasi, PhD

• Chen, Yiying Joyce, Alexion, AstraZeneca Rare Disease, Boston, Massachusetts, United States

Yiying Joyce Chen, MS

Background

Atypical hemolytic uremic syndrome (aHUS) is a rare thrombotic microangiopathy (TMA) caused by uncontrolled terminal complement activation. aHUS can be severe, requiring intensive care unit (ICU) support, yet outcome data are limited in those needing ICU care before starting ravulizumab (RAV), a complement C5 inhibitor (C5i).

Methods

This post hoc analysis examined patients who received ICU care before initiating RAV from the phase 3 single-arm clinical trial of C5i-naive adults with aHUS (NCT02949128). The primary endpoint was complete TMA response, comprising platelet count and lactate dehydrogenase normalization, and ≥25% improvement in serum creatinine from baseline (BL). The primary analysis period was up to week (W) 26; data were collected up to 4.5 years after RAV initiation (end of study [EOS]). Secondary endpoints included estimated glomerular filtration rate (eGFR) and dialysis status.

Results

The analysis included 27 patients (48.2% of trial population; 7 male, 20 female). Mean ages at RAV initiation and first aHUS symptom onset were both 42.0 (standard deviation [SD]: 16.5) years. Patients spent a mean of 10 (SD: 10) days in ICU before starting RAV. At BL, 96.3% of patients had extrarenal symptoms.
In total, 19 patients (70.4%) achieved complete TMA response by W26 and 21 (77.8%) by EOS. Median time to achieve complete TMA response was 44 (95% confidence interval: 22–86) days. Mean (SD) eGFR at BL was 12.6 (10.6) mL/min/1.73m², increasing to 58.1 (33.3) by W26 and 64.4 (28.5) by EOS. At BL, 19/27 patients (70.4%) were receiving dialysis, and among those who remained in the study, 86.7% (n=13/15) discontinued dialysis by W26, which was stable through EOS. One patient discontinued the study and drug owing to an adverse event and two died owing to sepsis; the deaths were not considered treatment related by the study investigators. No meningococcal infections were reported.

Conclusion

Most patients with aHUS receiving ICU care before starting RAV achieved complete TMA response by W26 (sustained response through EOS), improved eGFR and the majority could stop dialysis. This study demonstrates that adults with aHUS who were in the ICU before commencement of RAV have similar clinical outcomes to the overall aHUS trial population.

Acknowledgment

Writing assistance was provided by Elena Sugrue PhD of Oxford PharmaGenesis, Oxford, UK, and funded by Alexion, AstraZeneca Rare Disease.

Funding

• Commercial Support – Alexion, AstraZeneca Rare Disease.