Ravulizumab in adults and children with atypical hemolytic uremic syndrome: a plain language summary of three studies.

Abstract

This summary gives an overview of three published articles that report the results of research studies of ravulizumab, an approved treatment for people with atypical hemolytic uremic syndrome (often shortened to aHUS). This is a rare and serious condition where blood clots form in small blood vessels. Blood vessels are structures that transport blood around the body. Blood clots are the body's way of stopping someone from bleeding too much. However, if they form when they are not needed, they can cause harm. In atypical hemolytic uremic syndrome, the blood clots can cause injury to organs like the kidney. In the three studies, the researchers wanted to know if ravulizumab could decrease the formation of these clots and improve kidney function. Children who had never received ravulizumab or a similar treatment took part in the first study. Adults who had never received ravulizumab or a similar treatment took part in the second study. In the third study, children whose disease was already controlled by a medication called eculizumab switched to ravulizumab. Ravulizumab is dosed less frequently than eculizumab. The researchers looked at kidney function and the levels of different blood components to see how well the treatment was working. They also monitored the adverse effects that participants experienced. Across the three studies, ravulizumab improved indicators of blood clotting in small vessels and improved kidney function in both children and adults. In addition, ravulizumab was similarly effective to eculizumab for children who were already receiving eculizumab and switched to ravulizumab. Overall, the adverse effects that people experienced with ravulizumab were manageable. These studies showed that ravulizumab is a treatment option for children and adults with aHUS. In addition, a switch to ravulizumab can be considered for children who are already responding well to eculizumab and would benefit from less frequent dosing. Clinical Trial Registration: NCT03131219, NCT02949128, NCT03131219 (ClinicalTrials.gov).