Article No. 312
23 January 2020
Patient registries for rare diseases like atypical HUS are sometimes held by a single institution, or under the auspices of a university or hospital. In certain nations there is an established disease-specific registry to encompass patients within their own country. Some national aHUS registries share data within a collaborative and international registry, allowing for more robust and in-depth study. In the case of atypical HUS, there is also an industry-sponsored global aHUS registry.
Conceived as a clinical trial which was launched in 2012, the aHUS global registry is registered with the identifier NCT01522183 with the official title An Observational, Non-Interventional, Multi-Center, Multi-National Study of Patients With Atypical Hemolytic-Uremic Syndrome (aHUS Registry). While Alexion Pharmaceuticals funds this global aHUS registry, it does not decide or control the data or its use. Instead, the registry has an independent Scientific Advisory Board or SAB for oversight, management, and scientific rigor. This international team of expert clinicians and aHUS researchers are joined by patient representatives. (FMI on collaboration between the registry’s SAB and aHUS patients, see: an aHUS Alliance article or Orphanet research publication about this topic.)
New patient representatives to the global aHUS registry are Jeff Schmidt (USA) and Margriet Eygenraam (Canada), who will serve jointly in a volunteer capacity following the superlative term of Len Woodward (UK) which recently ended. Questions that aHUS patients want to know about created the aHUS Global Patients’ Research Agenda. launched by the aHUS Alliance on Rare Disease Day 2019. Attending their first SAB meeting in December 2019, Jeff and Margriet asked registry questions on behalf of aHUS patients around the world. Here they present registry FAQs in collaboration with Donna Ames, the Clinical Project Lead for Global Registry Operations at Alexion Pharmaceuticals.
Global aHUS Registry: What’s it all about?
The Global aHUS Registry was created in April 2012 to collect efficacy and safety data and record information on the progression of disease in aHUS patients. Many aHUS patients are familiar with this aHUS Registry but there also has been a lot of confusion as well.
When Margriet Eygenraam (aHUS Canada, aHUS Alliance) and Jeff Schmidt (aHUS Alliance, Rest of the World) took over the role of SAB Patient Advocate Representative for the aHUS Registry from Len Woodward (aHUSUK, aHUS Alliance), one of the first things they decided to do was to answer some questions the aHUS community had about the Registry. Some of them have already been answered by Len Woodward in the May 24, 2016 aHUS Alliance blog Global aHUS Registry (Click HERE to read). Jeff and Margriet worked with the Clinical Project Lead of the Registry to come up with answers to the following basic questions. If you still have questions about the Registry after reading Len’s blog and Margriet and Jeff’s answers below, please feel free to ask us.
Who can sign up?
Anyone who has been diagnosed with aHUS can sign up. There is no gender or age requirement. But it is required that your physician/institution be contracted to participate. If your doctor is not participating, she/he can make a request to a participating doctor, for you to be included. But this is not guaranteed.
Here is a link to “Atypical Hemolytic-Uremic Syndrome (aHUS) Registry” on clinicaltrials.gov (Click HERE) . You can see what institutions are participating and what country they are located in.
How does someone sign up?
Signing up to the Registry is done through your physician who must be at a participating site. The physician will first discuss with you about what is expected of you when participating. And then you must sign a consent form.
Do you have to be on Soliris to be in it?
You do not have to be receiving (or have previously received) Soliris or Ultomiris to be in the Registry. You only need to have a diagnosis of aHUS.
Will patients on Ultomiris be included?
Yes. But currently there is no protocol for the collection of data specific to Ultomiris.
Is the Registry limited to certain countries?
Yes. Alexion chooses which countries and which institutions within them can participate. This is based on a confidential internal strategy. Those whose country does not have a participating site can ask their doctor to request for patient participation through a participating institution in a nearby country. But the acceptance is not guaranteed.
How does someone know if they are in?
Participation requires a discussion from a physician from an institution that is participating in the Registry. And then the patient needs to sign a consent form. If that has not occurred, you are not in the Registry. It is unlikely if your physician is not a member of a participating institution that you are in the Registry. But if you are unsure, please ask your physician.
What does someone do as a participant?
The participant will first sign a consent form with the participating physician. The institution that the physician belongs to will send a questionnaire to the patient every 6 months asking questions about how they are feeling. The data manager at the site will send your completed questionnaire to the aHUS Registry to be entered into the database. Your medical data are collected from your medical records and entered into the database for the past 6 months. This includes data about transplants, dialysis, Soliris/Ultomiris, plasmapheresis, changes, infections, any lab results, etc.
What is the purpose of the Registry?
A disease registry is used for tracking the clinical care and outcomes of a certain patient population. Most disease registries are used to support care management for groups of patients with one or more chronic diseases. The main purpose of the aHUS Registry is to collect information on the safety and effectiveness of Soliris (and Ultomiris, soon) in patients with aHUS. What is learned may help patients and physicians understand more about this disease.
How long is the Registry going to last?
The Registry gets re-assessed every year to allow for continual learning and answering of questions. The aHUS Registry will likely continue until 2025 or longer. There is a desire to watch and learn about Ultomiris for at least 5 years.
Who can request information from the Registry?
Anyone can ask for data. The Scientific Advisory Board (SAB) decides what is shared and with whom. The requests for data are usually from participating physicians or their staff. Publications are soon going to be shared in lay-language which should help us be able to see what our shared data is teaching the SAB and the medical community.
How do you request the data?
A requestor would complete a Concept Sheet and send it to Alexion’s Epidemiologist who will review it and run a feasibility analysis to make sure that the Registry has the right data to answer the question(s). There may be some back and forth to ensure it is understood what is being asked for. Once it is confirmed that the data exists, the Concept Sheet is submitted and reviewed by the SAB. The Concept Sheet is reviewed for scientific merit and novelty of the research. Sometimes a small request becomes a full manuscript with a SAB champion who works to get the data into a peer-reviewed research project.
What kind of information can you get from the Registry?
The Registry collects historical data (including family history) and current data on patients who are treated with Soliris (and soon Ultomiris) that is related to safety and effectiveness of these medications. Since the Registry includes patients not treated with Soliris or Ultomiris the experts are able to see differences in patient health to hopefully better understand the disease.