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Kidney Week

Abstract: SA-PO922

Characteristics and Outcomes of Patients with Atypical Hemolytic Uremic Syndrome Switching to Ravulizumab from Eculizumab: A Global Registry Analysis

Session Information

Category: Glomerular Diseases

  • 1402 Glomerular Diseases: Clinical, Outcomes, and Trials

Authors

  • Schaefer, Franz S., Heidelberg University Hospital, Heidelberg, Germany
  • Al-Dakkak, Imad, Alexion, AstraZeneca Rare Disease, Boston, Massachusetts, United States
  • Anokhina, Ekaterina, Alexion, AstraZeneca Rare Disease, Boston, Massachusetts, United States
  • Cohen, David J., Columbia University Medical Center, New York, New York, United States
  • Greenbaum, Larry A., Emory University School of Medicine and Children's Healthcare of Atlanta, Atlanta, Georgia, United States
  • Ariceta, María, Vall d'Hebron Hospital, Barcelona, Spain
Background

There are currently no real-world cohort data on clinical characteristics and outcomes in patients with atypical hemolytic uremic syndrome (aHUS) who switched to ravulizumab (RAV) from eculizumab (ECU). This study aimed to address this need.

Methods

This was a post-marketing, observational, non-interventional, multinational registry study of patients diagnosed with aHUS who switched to RAV from ECU up to 27-Mar-23 (NCT01522183).

Results

Overall, 60 patients (24 pediatric) were included from Germany (45%), UK (18%), USA (18%), Denmark (10%), Spain (5%), and Israel (3%). Median (range) age at RAV initiation was 34 (2–72) years and 70% of patients were female. Overall, 58% of patients had a pathogenic variant or anti-complement factor H antibodies. Median (range) time on treatment was 21 (2–40) months for RAV and 62 (11–155) months for ECU. No new events of dialysis, kidney transplant, or thrombotic microangiopathy (TMA) relapse were reported during RAV treatment. Estimated glomerular filtration rates (eGFR) remained stable during the evaluation period (Figure).

Conclusion

Switching to RAV from ECU resulted in sustained maintenance of kidney function without evidence of new events of dialysis, kidney transplant, or TMA relapse over the median treatment period of 21 months. These results support the real-world effectiveness of RAV in patients with aHUS who switched from ECU.

Funding

  • Commercial Support – Alexion, AstraZeneca Rare Disease, Boston, MA, USA.