A lack of patient engagement is an issue across many rare diseases, with atypical hemolytic uremic syndrome (aHUS) acting as the perfect example. In fact, aHUS patient organizations are still running into the same obstacles they were facing a decade ago. This means that there is a lot of room for improvement.
The aHUS Alliance lists a number of barriers that stand in the way of patient engagement and have for a long time. These include:
- Minimal awareness and understanding of the issues faced by aHUS patients and their loved ones
- Projects and other efforts that were undertaken without input from aHUS patients or organizations
- A lack of effort and knowledge when it comes to aHUS patients and the larger community
- A general lack of understanding of aHUS in general
- This can lead to issues in surveys and other research efforts
- Assumptions that aHUS patients face the same issues as other rare disease patients
All of these obstacles stand in the way of patient engagement and are very frustrating for patients, their families, and advocates.
While these problems are in the way, patient interest is not one of them. In fact, a 2016 poll found that 60% of aHUS patients who participated had already taken part in research and would like to do so again. Another 36% wanted to participate in future research despite no prior experience.
The interest is there. Now we just need to include and listen to these patients. First up, it’s important to pay attention to aHUS news and issues from all around the world. Because there are different regulatory landscapes throughout different countries, it is necessary to stay up to date in each place. To learn more about the global agenda for aHUS patients, click here.
Continuing to listen to patients, you should utilize resources that were created by aHUS patients or advocacy organizations. Go straight to the source to learn about this disease and patient experiences. Here is a great resource from the aHUS Alliance, and it’s the perfect place to start.
Moving forward, we should also be listening to patient leaders. These people hold so much knowledge and insight on aHUS, the patient experience, and patient engagement. They are an amazing resource to utilize.
In the end, it’s important to recognize the hard work and dedication behind volunteering and advocacy and give the proper appreciation. It’s often a thankless, rewardless job, and the least we can do is give these people the respect they deserve.
Atypical hemolytic uremic syndrome is a rare kidney function disorder. It causes fatigue, irritability, high blood pressure, thrombocytopenia, hemolytic anemia, blood in the urine, and acute kidney failure. All of these symptoms are the result of a genetic predisposition in addition to an environmental trigger. Common events that set off aHUS include the flu, chickenpox, or pregnancy. Diagnosis of this condition can be difficult, as it is very rare and faces low awareness. Doctors will look for malfunctioning kidneys, hemolytic anemia, a low platelet count, and known genetic mutations. After a diagnosis is established, doctors may recommend plasma therapy, blood transfusions, eculizumab, medications to expand blood vessels, blood pressure drugs, and other therapies.