If you have atypical hemolytic uremic syndrome (aHUS), you may want to consider enrolling in the ongoing global aHUS registry study still recruiting patients.
Information collected through this registry will, among other uses, help researchers to better understand, diagnose, and treat this rare disease.
What is aHUS?
aHUS is marked by blood clots forming in the small blood vessels of the kidneys. Clots can damage these essential organs by restricting blood flow, and lead to health problems that can include hemolytic anemia, thrombocytopenia, kidney failure, shortness of breath, nausea, fatigue, heart disease, and seizures.
Alexion Pharmaceuticals sponsors the aHUS registry (NCT01522183), which opened in 2012 and runs through December 2023. It plans to follow up to 2,000 patients, enrolled at 128 locations across 15 countries, for up to 10 years.
Alexion designed this observational registry study to collect post-marketing clinical data on the long-term safety of its two approved aHUS treatments, Soliris (eculizumab) or Ultomiris (ravulizumab).
People with aHUS who are not using either treatment are also welcome to enroll. Data across groups of patients will help in better understanding disease progression.
As an observational study, registry participants will not be given any treatment — approved or experimental — and, again, do not need to be using a given treatment to take part.
As of Nov. 10, 2020, sites in the U.S., Canada, Australia, and a number of European countries are no longer recruiting patients.
Enrollment may be ongoing at sites across Belgium, France, Poland, and South Korea; contact and site location information is available by scrolling through this list.
What are the registry’s goals?
Researchers designed the registry to capture real-world data over time on aHUS patients, including their treatments, symptoms, and overall health.
Specifically, the study aims to assess the long-term manifestations of thrombotic microangiopathy, or the formation of blood clots in the body’s small blood vessels.
It will also measure other clinical outcomes, including morbidity and mortality, in aHUS patients using Soliris, Ultomiris, or other treatments.
By collecting this information from people worldwide, scientists can learn more about disease treatment, outcomes and how best to manage this disease.
Data can also help in educating physicians, many of whom likely are not be familiar with this rare disease, about its signs and symptoms in people they are treating. The earlier a person is correctly diagnosed with a given disease, the earlier treatment can begin.
Who can participate?
The registry is open to people of any age with a clinical aHUS diagnosis — boys and girls, men and women. Patients, or a parent or legal guardian of a minor, must give written consent.
How does the registry work?
Study centers are responsible for patient enrollment, supplying the registry with basic medical information and data, and updating that information at regular intervals.
Every six months, enrolled patients are asked to complete a questionnaire about their physical health and overall well being. A goal is to learn how living with aHUS, and its treatments affect patients in their day-to-day life.
The registry’s independent scientific advisory board, which is composed of leading aHUS researchers, controls how the data are used.
Last updated: Nov. 9, 2020
aHUS News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
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