Abstract: FR-PO0671
Long-Term Real-World Effectiveness of Ravulizumab Among C5-Inhibitor-Naive Patients with Atypical Hemolytic Uremic Syndrome (aHUS): A Physician-Panel-Based Chart Review Study (aHUS-EXTEND Study)
Session Information
- Glomerular Diseases: Clinical, Outcomes, and Therapeutics Research - ANCA/FSGS
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
- Hanna, Ramy Magdy, University of California, Irvine, California, United States
- Kaufeld, Jessica Katharina, Hannover Medical School, Hannover, Germany
- Wang, Yan, Alexion, AstraZeneca Rare Disease, Boston, Massachusetts, United States
- Nag, Arpita, Alexion, AstraZeneca Rare Disease, Boston, Massachusetts, United States
- Kunjappu, Mary, Alexion, AstraZeneca Rare Disease, Boston, Massachusetts, United States
- Huynh, Lynn, Analysis Group, Inc, Boston, Massachusetts, United States
- Burdeau, Jordan, Analysis Group, Inc, Boston, Massachusetts, United States
- Young-Xu, Leili, Analysis Group, Inc, Boston, Massachusetts, United States
- Duh, Mei Sheng, Analysis Group, Inc, Boston, Massachusetts, United States
- Chaturvedi, Shruti, Johns Hopkins University, Baltimore, Maryland, United States
- Gaeckler, Anja H., University Hospital Essen, Essen, Germany
Background
Atypical hemolytic uremic syndrome (aHUS) is a rare thrombotic microangiopathy (TMA) driven by complement dysregulation. Ravulizumab (RAV), a C5 inhibitor (C5i), is approved for the treatment of aHUS based on registrational clinical trials; further long-term real-world evidence is warranted.
Methods
This retrospective, longitudinal, physician-panel-based chart review included C5i-naive adult patients with aHUS in the USA and Germany treated with RAV. Physicians with medical and laboratory records for ≥ 1 patient with aHUS randomly selected 1–5 patients with ≥ 36 months of follow-up after RAV initiation, unless the patient had died. The index date was defined as date of RAV initiation. Outcomes assessed included laboratory parameters, complete TMA response (a composite hematologic/renal endpoint) and dialysis status. End-stage kidney disease (ESKD) was defined as a diagnosis of chronic kidney disease with an estimated glomerular filtration rate below 15 mL/min/1.73 m2.
Results
In total, 124 patients were analyzed (USA: n=88; Germany: n=36). No patients had undergone kidney transplantation before RAV initiation. The mean duration of follow-up was 4.1 (standard deviation: 0.9) years. By the end of follow-up, 76 patients (61.3%) remained on RAV; 48 patients (38.7%) discontinued RAV after a median (interquartile range) treatment duration of 13.0 (8.2–33.1) months. Overall, 84 patients (67.7%) achieved complete TMA response within 12 months post-index, increasing to 120 patients (96.8%) by end of follow-up. At 36 months, the mean reduction in serum creatinine was 53.3%. Among 43 patients receiving dialysis at baseline, 33 (76.7%) were able to discontinue dialysis during follow-up. Of the 80 patients not requiring dialysis at baseline, one (1.3%) initiated dialysis during follow-up. Over the follow-up period, 3 patients (2.4%; 1 USA; 2 Germany) met ESKD criteria, and no patients received kidney transplantation.
Conclusion
This study provides real-world evidence of long-term improvements in kidney function with continued RAV therapy, including a substantial proportion of patients achieving freedom from dialysis following treatment with RAV.
Acknowledgment
Writing assistance was provided by Innes Jarmson PhD of Oxford PharmaGenesis, Oxford, UK, and funded by Alexion, AstraZeneca Rare Disease.
Funding
- Commercial Support – Alexion, AstraZeneca Rare Disease