Ravulizumab improves kidney function in patients with atypical hemolytic uremic syndrome
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Key takeaways:
- Researchers reviewed the efficacy of ravulizumab based on 2-year data from two phase 3, single-arm studies.
- eGFR was maintained in pediatric and adult patients throughout the study period.
Ravulizumab was successful in improving blood health, kidney function and quality of life while maintaining eGFR in pediatric and adult patients with atypical hemolytic uremic syndrome, according to published data.
“These results support ravulizumab as a long-term treatment for people with [atypical hemolytic uremic syndrome] aHUS,” Bradley P. Dixon, MD, FASN, professor of pediatrics and medicine and head of the renal section of the department of pediatrics at the University of Colorado School of Medicine, and colleagues wrote in Kidney Medicine.
“aHUS is a rare genetic disease that is caused by the inability of the body’s complement system to be properly regulated,” Dixon told Healio. “The endothelial cells become injured by this uncontrolled complement activation, and this causes tiny clots to form in the capillaries of the body causing a consumption of platelets and low platelet counts in the blood; hemolysis as the red blood cells are passing through the capillaries containing the tiny clots and organ dysfunction including kidney dysfunction from the clots choking off blood supply and oxygen and nutrients that it carries,” he said.
“This condition looks clinically very similar to the [Escherichia coli] E. coli (Shiga toxin) form of hemolytic uremic syndrome caused by food poisoning from undercooked food but is a very different form of HUS.”
Ravulizumab, a complement C5 inhibitor (C5i) approved for the treatment of aHUS, turns off the complement system and stops the injury to endothelial cells, Dixon said. “Ravulizumab was developed from an earlier form of the complement-blocking drug eculizumab, but has a much longer duration in the body, so fewer/less frequent doses of ravulizumab have to be given compared to eculizumab to control the disease,” Dixon told Healio.
Dixon and colleagues analyzed data from two clinical trials involving children and adults with aHUS who received ravulizumab intravenously every 4 to 8 weeks, depending on patient weight. One study included C5i-naive adults, and the second study included two cohorts of pediatric patients (C5i-naive and those who switched to ravulizumab from eculizumab).
“The primary endpoint in the studies of C5i-naive patients was complete [thrombotic microangiopathy] TMA response, which consisted of platelet count normalization, lactate dehydrogenase normalization and [at least] 25% improvement in serum creatinine from baseline, at two consecutive assessments at least 4 weeks apart,” the researchers wrote.
“Complete TMA response rates over 2 years were 61% and 90% in C5i-naive adults and pediatric patients, respectively,” the researchers wrote. “The median increase in eGFR from baseline was maintained over 2 years in C5i-naive adults (35 mL/min/1.73 m2) and pediatric patients (82.5 mL/min/1.73 m2).”
The researchers wrote that quality of life scores improved for patients who had to take fewer treatment doses compared with eculizumab, and no meningococcal infections were reported.
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