Global aHUS Registry

Global aHUS Registry
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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.

The registry

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

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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.

Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours d’Elegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled “Belle of the Concours.”

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Özge has a MSc. in Molecular Genetics from the University of Leicester and a PhD in Developmental Biology from Queen Mary University of London. She worked as a Post-doctoral Research Associate at the University of Leicester for six years in the field of Behavioural Neurology before moving into science communication. She worked as the Research Communication Officer at a London based charity for almost two years.
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Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours d’Elegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled “Belle of the Concours.”

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