New study supports use of Soliris as first-line pediatric aHUS treatment
Real-world data show approved therapy safe, effective in children with HUS
Soliris (eculizumab) is safe and effective for treating children with various types of hemolytic uremic syndrome (HUS), including atypical HUS (aHUS), according to a real-world study involving pediatric patients in Israel.
In fact, more than half of the children with aHUS given Soliris in the study achieved a complete response, meaning they showed sustained normalization of blood parameters and kidney function improvements.
Overall, the results support the use of Soliris as a first-line treatment for pediatric patients with aHUS, and indicate its “potential efficacy beyond [its] primary indication” in other HUS types, the researchers noted.
In the U.S., Soliris is approved for use in adults and children with aHUS.
The study, “Eculizumab is efficacious and safe in pediatric patients with various forms of hemolytic uremic syndrome: a retrospective clinical experience of a tertiary center,” was published in the journal Frontiers in Pharmacology.
Over half of aHUS pediatric patients given Soliris show complete response
In aHUS, certain genetic mutations result in the uncontrolled activation of the complement cascade, a group of proteins that work with the immune system to protect the body against infections. This improper activation can lead to thrombotic microangiopathy, or TMA, where blood clots form in small blood vessels, blocking blood flow and causing organ damage.
The other hallmark symptoms of aHUS include the destruction of red blood cells, known as hemolytic anemia, and low platelet counts, or thrombocytopenia.
Soliris is an antibody that works by targeting a complement protein called C5, thus preventing the complement cascade from becoming overly active. In the U.S., Soliris and several biosimilars are approved to inhibit complement-mediated TMA in adults and children with aHUS.
In this study, researchers from several institutions in Israel looked at how safe and effective Soliris is in pediatric patients with various types of HUS, including aHUS. To do this, the team reviewed the medical records of 24 children treated with the medication at an Israeli hospital between 2011 and 2020.
The children had a median age of 5.8 years. Among them were 13 diagnosed with aHUS, five with a type of HUS caused by Escherichia coli bacteria (STEC-HUS), and six who had developed TMA after receiving a transplant. All were followed for up to 28 weeks, or slightly longer than six months, after starting treatment with Soliris.
Half of the children — including seven with aHUS — achieved a complete TMA response, defined as a sustained normalization of blood parameters and improvements in kidney function. Moreover, nearly two-thirds (63%) remained free from new TMA events over the 28 weeks.
Efficacy was demonstrated overall, and no severe adverse events were attributed to the drug. … Early intervention [with Soliris] was more effective across all HUS indications.
Many of the children also showed improvements in lab tests: 63% had higher platelet counts; 76% had lower levels of lactate dehydrogenase, a marker of cell damage; 60% had higher levels of hemoglobin, which is the protein that carries oxygen in the blood; and 79% had better kidney function.
No severe side effects were reported with Soliris. Four children died, but none of the deaths were related to treatment.
“Efficacy was demonstrated overall, and no severe adverse events were attributed to the drug,” the researchers wrote. The team also noted that “early intervention [with Soliris] was more effective across all HUS indications.”
According to researchers, the safety and efficacy of Soliris in this study “potentially expands its therapeutic applications.”
Overall, the findings confirm the use of Soliris as a first-line treatment for aHUS, the researchers noted. Further, per the team, the response seen in STEC-HUS patients suggests possible treatment effectiveness beyond Soliris’s primary indication.
“Well-designed, prospective, randomized controlled trials are needed, encompassing a larger, more diverse patient population, with varying disease severities,” the team concluded.
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