Abstract: PO1655
Early Eculizumab Withdrawal in Atypical Hemolytic Uremic Syndrome Is Safe and Cost-Effective
Session Information
- Glomerular Diseases: Treatment and Outcomes
November 04, 2021 | Location: On-Demand, Virtual Only
Abstract Time: 10:00 AM - 12:00 PM
Category: Glomerular Diseases
- 1203 Glomerular Diseases: Clinical, Outcomes, and Trials
Authors
- Bouwmeester, Romy N., Radboudumc, Nijmegen, Gelderland, Netherlands
- Duineveld, Caroline, Radboudumc, Nijmegen, Gelderland, Netherlands
- Wijnsma, Kioa L., Radboudumc, Nijmegen, Gelderland, Netherlands
- Van Den Heuvel, L.P.W.J., Radboudumc, Nijmegen, Gelderland, Netherlands
- Wetzels, Jack F., Radboudumc, Nijmegen, Gelderland, Netherlands
- Van De Kar, Nicole, Radboudumc, Nijmegen, Gelderland, 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, a national observational study monitoring eculizumab discontinuation in Dutch aHUS patients after three months of therapy.
Methods
All pediatric and adult aHUS patients with native kidneys and first-time eculizumab treatment (n=21) were evaluated. At last, 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 After a treatment duration of 13.6 weeks (range 2.1-43.9), eculizumab was withdrawn in all patients (Figure 1). 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 clinically relevant predictors of relapse (including the presence of a pathogenic mutation) could be determined. The total medical expenses, including costs of among others hospital admission and disease recurrence, of our population were only 33% 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.
Outcomes of CUREiHUS patients