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Abstract: TH-PO502

Long-Term Outcomes in Eculizumab-Treated Patients Enrolled in the Global aHUS Registry

Session Information

Category: Glomerular Diseases

  • 1303 Glomerular Diseases: Clinical‚ Outcomes‚ and Trials

Authors

  • Greenbaum, Larry A., Division of Pediatric Nephrology, Emory University School of Medicine and Children's Healthcare of Atlanta, Atlanta, Georgia, United States
  • Al-Dakkak, Imad, Alexion, AstraZeneca Rare Disease, Boston, Massachusetts, United States
  • Anokhina, Ekaterina, Alexion, AstraZeneca Rare Disease, Zurich, Switzerland
  • Ardissino, Gianluigi, Center for HUS Prevention Control and Management, Fondazione IRCCS Ca' Granda-Ospedale Maggiore Policlinico, Milan, Italy
  • Ariceta, Gema, Paediatric Nephrology Department, University Hospital Vall d'Hebron, Barcelona, Spain
  • Kumar, Gurinder, Department of Paediatrics, Sheikh Khalifa Medical City, Abu Dhabi, United Arab Emirates
  • Licht, Christoph, Division of Nephrology, The Hospital for Sick Children, Toronto, Ontario, Canada
  • Siedlecki, Andrew M., Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts, United States
  • Vande walle, Johan, Pediatric Nephrology Unit, Ghent University Hospital, Ghent, Belgium
Background

The global atypical hemolytic uremic syndrome (aHUS) Registry (NCT01522183) is the largest repository of real-world data on patients with aHUS. We report long-term (LT) outcomes with eculizumab (ecu) treatment in two patient cohorts: those with LT follow-up (FU) without progression to end-stage kidney disease (ESKD) and those who developed ESKD during treatment. Our aim was to determine whether LT ecu preserves kidney function.

Methods

The analysis included registry patients enrolled between Apr 2012–Nov 2021 and treated with ecu for ≥90 days. Cohort A included patients with ≥3-year FU, no ESKD, and two creatinine values; near treatment start and after ≥3-year observation. Cohort B included patients who developed ESKD after treatment with no FU duration specified.

Results

Demographics and laboratory parameters (baseline [BL], last FU [LFU]) are shown in the Table. For Cohort A, 249 patients met inclusion criteria; at LFU, eGFR improved, 7% received acute dialysis (9% of adults, 6% of children) and two adults died. For Cohort B, 56 patients met inclusion criteria; at LFU, 48% received a kidney transplant (38% of adults, 68% of children), 89% received dialysis (95% of adults, 79% of children) and eight died (six adults, two children). BL eGFR was lower in patients who progressed to ESKD (Cohort B, N=56) than in patients who did not progress (Cohort A, N=249). Platelet and LDH levels improved in both cohorts at LFU.

Conclusion

In a real-world setting, eGFR was stable in most patients receiving ecu. Some patients demonstrated hematologic benefits but no renal response; they had lower BL eGFR and initiated treatment late.

Funding

  • Commercial Support