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Abstract: TH-PO503

Early Eculizumab Withdrawal in Patients With Atypical Hemolytic Uremic Syndrome in Native Kidneys Is Safe and Cost-Effective: Results of the CUREiHUS Study

Session Information

Category: Glomerular Diseases

  • 1303 Glomerular Diseases: Clinical‚ Outcomes‚ and Trials

Authors

  • Bouwmeester, Romy N., Radboud UMC, Nijmegen, Netherlands
  • Duineveld, Caroline, Radboud UMC, Nijmegen, Netherlands
  • Wijnsma, Kioa L., Radboud UMC, Nijmegen, Netherlands
  • Adang, Eddy M., Radboud UMC, Nijmegen, Netherlands
  • Van den heuvel, Lambertus P.W.J., Radboud UMC, Nijmegen, Netherlands
  • Wetzels, Jack F., Radboud UMC, Nijmegen, Netherlands
  • Van De Kar, Nicole, Radboud UMC, Nijmegen, Netherlands

Group or Team Name

  • on behalf of the CUREiHUS study group
Background

The introduction of eculizumab has improved outcome in patients with atypical hemolytic uremic syndrome (aHUS). The optimal treatment strategy is debated. It is unknown if unbiased withdrawal of eculizumab is a safe strategy. Here we report the results of the CUREiHUS study, an observational study monitoring eculizumab discontinuation in Dutch aHUS patients after 3 months of therapy.

Methods

All pediatric and adult aHUS patients with native kidneys and first-time eculizumab treatment (n=21) were evaluated. In addition, an extensive cost-effective analysis was conducted.

Results

In the period from January 2016 till October 2020 we included 21 patients. All patients showed full recovery of hematological thrombotic microangiopathy (TMA) parameters after start of eculizumab. A renal response was noted in 18 patients. After a treatment duration of 13.6 weeks (range 2.1-43.9), eculizumab was withdrawn in all patients. During follow-up (80.7 weeks (0.0-236.9)), a relapse occurred in four patients (19.0%). Median time to first relapse was 14.3 weeks (7.1-62.0). Eculizumab was re-initiated within 24 hours in all relapsing patients. At last follow-up, there were no chronic sequelae, i.e. no clinically relevant increase in serum creatinine, proteinuria and/or hypertension, in the relapsing patients. No predictors of relapse could be determined. The total medical expenses of our population were only 30% of the fictive expenses made when patients would have received eculizumab every fortnight.

Conclusion

It is safe and (cost-)effective to discontinue eculizumab after three months of therapy in patients with aHUS in native kidneys. Larger data registries are needed to determine factors to predict relapse(s) and short- and long-term outcomes.