Abstract
What is this summary about?
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.
What were the results?
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.
What do the results mean?
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.
Keywords: aHUS, atypical hemolytic uremic syndrome, blood clots, complement system, dialysis, eculizumab, ravulizumab
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Acknowledgments
The authors thank all study participants, investigators, and staff as well as the authors of the three original publications.
Footnotes
Financial & competing interests disclosure
M Nowicki has received consulting and speaking fees from AstraZeneca, Sanofi, Amicus, Swixx, Chiesi and Boehringer Ingelheim; and congress sponsorship from Amicus and AstraZeneca. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
Competing interests disclosure
The authors have no competing interests or relevant affiliations with any organization or entity with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.
Writing disclosure
Dr Pablo Rivas helped the authors write this summary on behalf of Content Ed Net.
Open access
This work is licensed under the Attribution-NonCommercial-NoDerivatives 4.0 Unported License. To view a copy of this license, visit http://creativecommons.org/licenses/by-nc-nd/4.0/