Ultomiris protects transplanted kidneys in real-world study
Therapy improved vital blood markers and sustained new organs
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Ultomiris (ravulizumab) is a safe and effective way to prevent disease recurrence in people with atypical hemolytic uremic syndrome (aHUS) who undergo a kidney transplant, a real-world study in Spain shows.
Data from 68 adult patients found that six months of treatment led to significant improvements in blood markers and kidney function. These benefits were especially pronounced in patients who received Ultomiris as a preventive measure right after their transplant, and the therapy successfully maintained disease control for those who switched over from Soliris (eculizumab).
According to researchers, “these findings support [Ultomiris] use as a valid option for first-line treatment of aHUS in kidney transplantation.” They also show the treatment maintained “disease control with extended dosing intervals” in patients who switched from Soliris.
The study, “Ravulizumab in Renal Transplantation: Results of a Spanish Multicenter Study,” was published in the journal Nefrología.
The role of complement inhibition in kidney transplants
aHUS is caused by abnormal activity of the complement cascade (a part of the immune system) that leads to blood clotting in small blood vessels, or thrombotic microangiopathy. The disease is marked by kidney damage, low levels of platelets (tiny cell fragments that help the blood clot), and the destruction of red blood cells, known as hemolysis.
Most people with aHUS have mutations in genes encoding proteins of the complement cascade, but a triggering event such as an infection is typically needed to develop the disease.
Ultomiris is a complement pathway inhibitor designed to prevent blood clot formation in people with aHUS. However, there has been limited data on its use in patients undergoing kidney transplants.
In the study, researchers conducted a retrospective analysis to assess the safety and efficacy of Ultomiris in 68 adults diagnosed with aHUS before or after a kidney transplant, treated at 14 hospitals across Spain. Patients had a mean age of 46.8 years. Most underwent a single kidney transplant (64.7%).
Genetic testing was available for all participants. The most common disease-associated variants affected the gene encoding complement factor H, found in 35.3% of patients. Overall, nearly two-thirds of patients were classified as having a high risk of aHUS recurrence.
Ultomiris was more frequently (75%) given after patients switched from Soliris, while others received the treatment to prevent aHUS episodes (20.6%) or to treat a new aHUS episode (4.4%). The switch from Soliris to Ultomiris was likely driven by Ultomiris’ lower infusion frequency and link to a better quality of life, according to the researchers. Of note, the two therapies are marketed by Alexion, AstraZeneca Rare Disease, which funded medical writing and reviewed the data for this study.
After six months of treatment, no aHUS recurrences were reported in the overall group of participants. Several key laboratory markers improved, including hemoglobin, which is the protein that carries oxygen in red blood cells, and platelet levels. Kidney function also improved, reflected by lower creatinine and improved estimated glomerular filtration rates. These benefits were also seen in the subgroups of those who received Ultomiris as a preventive treatment and patients taking it for a new aHUS episode.
In patients who switched from Soliris, whose mean time between their kidney transplant and starting on Ultomiris was 43.5 months (slightly more than 3.5 years), blood parameters and kidney function remained largely stable over time. According to the researchers, these findings reinforce the efficacy of Ultomiris in maintaining disease control.
Kidney function appeared to be better among patients who received a kidney transplant from a living donor than among patients who received a kidney transplant from a donor who had died. No significant differences in aHUS recurrence risk were seen.
Ultomiris was generally well tolerated, with no cases of meningitis — an inflammation of the membranes that surround the brain and spinal cord — or loss of the transplanted kidney. No infusion-related side effects or infections leading to treatment discontinuation were reported. No patients stopped the treatment during the study.
The most common infections were urinary tract infections, affecting about one-quarter of patients. Cytomegalovirus infections were reported in 11 participants, mostly among those who received Ultomiris as preventive treatment. Infections by the BK virus, usually inactive in the body, occurred in 10 patients, particularly among those who switched to Ultomiris.
“Overall, our study findings support the efficacy and safety of [Ultomiris] in patients with aHUS who have received a kidney transplant. Additionally, its extended dosing interval may reduce healthcare burden and improve quality of life, specifically among transplant recipients requiring lifelong treatment,” the researchers wrote.
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