This is a plain language summary of the final article to be published about Stopping Eculizumab Treatment Safely Clinical Trial.
The aim of the Stopping Eculizumab Treatment Safely atypical haemolytic uraemic syndrome trial was to determine whether an alternative way to treat atypical haemolytic uraemic syndrome was feasible and safe. This included treatment with eculizumab to control disease, withdrawal of treatment and reintroduction if needed. Twenty-eight patients stopped eculizumab and 11 patients continued treatment. We interviewed 20 patients or parents to explore experiences of living with atypical haemolytic uraemic syndrome and of participating in the trial.
What did we find?
Overall, 24 patients remained off eculizumab during their 2 years of trial participation. Four patients relapsed and eculizumab treatment was re-started. One patient’s kidney function fell, but overall withdrawing eculizumab had a similar safety profile as staying on treatment. The cause of atypical haemolytic uraemic syndrome predicted relapse.
The quality of life of patients who withdrew from eculizumab is expected to increase, with minimal impact on life expectancy. We estimated that there would be a saving of £4,188,361 per patient who withdrew from treatment.
In the interviews at the beginning of the trial, participants said that eculizumab treatment was disruptive to work and school. Eculizumab side-effects were also a problem. The decision not to withdraw from eculizumab was based upon concerns about the possibility of relapse. At the end of the trial, participants said that the extreme tiredness and other symptoms associated with eculizumab were no longer present with less disruption to their daily lives. Anxiety was an issue, but this became less over time. Knowing they could restart eculizumab immediately was reassuring.
What next?
The following future research is important: (1) Can eculizumab be withdrawn again in patients who have relapsed? (2) What are the factors influencing risk of relapse? (3) What is the most effective way to manage patients short and long-term after withdrawal of eculizumab? It will be critical to identify what resources are required to implement new ways to manage atypical haemolytic uraemic syndrome.
The full article can be read HERE.
Previous patients’ article HERE
Article No 784
