It’s Safe to Stop Soliris for aHUS Children in Remission: Study
But close monitoring is still needed due to possible recurrence
Soliris (eculizumab) can safely be discontinued in children with atypical hemolytic uremic syndrome (aHUS) whose disease is in remission, but close monitoring is necessary due to the possibility of aHUS recurrence, a recent study in the Arabian Gulf region highlights.
The study found that more than 70% of patients remained in remission after stopping treatment. But just over one-quarter of the children, or 28.5%, “experienced HUS relapse after discontinuation, making the identification of at-risk patients essential,” the researchers wrote.
“Resuming eculizumab immediately after relapse is crucial and effective in most patients,” the team added.
This study was conducted across five countries in the Arabian Gulf region.
Investigating treatment discontinuation
Titled “A Multicenter Study Evaluating the Discontinuation of Eculizumab Therapy in Children with Atypical Hemolytic Uremic Syndrome,” the study was published in the journal Children.
Soliris is a widely approved treatment for aHUS that is administered intravenously through regular infusions into the bloodstream. It is given every week for a month, and then every other week thereafter.
Prescribing information for Soliris does not specify how long treatment should be continued once aHUS is in remission. Theoretically, the therapy could be maintained indefinitely, but that would be costly and also increases the risk of long-term side effects.
Thus, researchers are now examining whether the treatment can safely be stopped — and working to figure out best practices for discontinuation.
Here, an international team of scientists conducted a review of data from 28 children with aHUS who were given Soliris, and then discontinued treatment.
The children were treated at five centers in Saudi Arabia, two centers in the United Arab Emirates (UAE), and one each in Oman, Qatar, and Kuwait.
The patients had a mean age of 3.47 years and about 70% were male. Nearly two-thirds had required dialysis prior to starting treatment with Soliris.
According to the researchers, the children were on Soliris for a median of one year, and were followed for a median of two years after stopping treatment.
After taking Soliris, the majority of patients — 23 of 28, or 82.1% — were in complete remission from aHUS, the researchers reported. Disease remission was the most common reason listed for stopping treatment; other reasons given included negative genetic testing and family preference.
After stopping treatment with Soliris, eight patients — the 28.5% — experienced an aHUS relapse. The median time between stopping Soliris and disease relapse was three months, and the most common triggering factor was an upper respiratory tract infection.
Statistical analyses found the risk of relapse was significantly higher among patients in whom an aHUS-associated mutation was detected.
Other factors, including age, sex, need for dialysis, and kidney function measures, all showed no association with relapse risk. There also was no correlation between relapse risk and the duration of Soliris treatment prior to discontinuation.
All of the patients who relapsed restarted treatment, and all were in remission following renewed Soliris therapy. Only one patient developed new chronic kidney disease after discontinuing the therapy.
“The outcome after discontinuation in this study was fairly good, with the majority of children remaining in remission with normal kidney function,” the researchers wrote.
“Although relapses happened, resuming [Soliris] therapy was effective in controlling the disease,” they added.
The team concluded that Soliris can safely be discontinued in children with aHUS who are in remission, but noted the necessity of monitoring patients for signs of relapse. They also stressed a need for future studies to clarify what factors affect the risk of disease recurrence after Soliris discontinuation.
“A full assessment of the risk factors before the decision to discontinue [Soliris] is essential, with parents being involved in the decision,” the team wrote.
Soliris is marketed by AstraZeneca, which was not involved in this study.
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