Real-world evidence from the Global aHUS Registry confirms the safety and effectiveness of switching to ravulizumab from eculizumab in patients with aHUS: a plain language summary

Abstract

Registry; Atypical hemolytic uremic syndrome; Ravulizumab

Registre; Síndrome hemolític urèmic atípic; Ravulizumab

Registro; Síndrome hemolítico urémico atípico; Ravulizumab

What is aHUS? Atypical hemolytic uremic syndrome (aHUS) is a rare disease that is caused by tiny blood clots that can block small blood vessels in the body, especially in the kidneys. This happens when a part of the immune system, called the complement system, becomes overactive. What are ravulizumab and eculizumab? Ravulizumab and eculizumab are approved treatments that help to prevent overactivity of the complement system by blocking a protein called complement component C5. Ravulizumab is derived from eculizumab and works immediately to block C5. It also lasts longer than eculizumab so can be given less often (every 4–8 weeks compared with every 2 weeks with eculizumab). What is the current gap in knowledge in aHUS? There are limited data on how well ravulizumab works to treat aHUS based on real-life patient experiences. What did this study look at? This article describes a study on the real-world safety and effectiveness of ravulizumab in patients with aHUS who switched from eculizumab, using data from the Global aHUS Registry, which has been collecting information since April 2012. Who was involved in the study? The study included 60 patients (43 adults and 17 children) who switched to ravulizumab from eculizumab, including 15 who received a kidney transplant before starting ravulizumab treatment. What were the safety results? Overall, 13 patients experienced an unwanted reaction or side effect after ravulizumab treatment, but none of these were unexpected by their doctors. Three of these reactions (fatigue and headache in 1 patient and infusion reaction in another patient) were thought to be related to ravulizumab treatment. There were no cases of meningococcal infection (a serious bacterial infection that may be related to treatment with either ravulizumab, eculizumab or another treatment that affects the complement system) or deaths during treatment. What were the effectiveness results? A subset of patients (49 out of 60) was eligible for the effectiveness analyses. These were patients who were treated with ravulizumab long enough to assess how well it works and who had a short time gap between switching to ravulizumab from eculizumab. No patient required dialysis or kidney transplantation while receiving ravulizumab. Markers of disease measured by blood tests were stable after switching to ravulizumab from eculizumab. What do the results mean? The study provides real-world evidence that ravulizumab is safe and works in patients with aHUS after switching from eculizumab.

The Global aHUS Registry is sponsored by Alexion, AstraZeneca Rare Disease. This plain language summary was sponsored by Alexion, AstraZeneca Rare Disease.