What Do Most HAE Patients Want From Treatment? Fewer Attacks, Survey Finds

The vast majority of people with hereditary angioedema (HAE) stress the importance of their preventative therapy correcting C1 inhibitor protein deficiency, allowing for fewer attacks, as the leading factor in evaluating a prophylactic treatment, according to a survey by The Harris Poll.

Safety considerations followed closely in importance.

The online survey, sponsored by CSL Behring, engaged 100 adults with HAE along with 150 licensed allergists and immunologists in the U.S. who have treated at least two people living with HAE in the 12 months before the survey. 

A total of 91% of the physicians also agree that fewer attacks and safety are the most critical factors when prescribing prophylactic therapy. However, 72% of patients strongly agree that convenience in treatment — like fewer injections — is of lesser importance than fewer attacks, while 28% of physicians express similar views.

“This data shows that those living with HAE [94%] and their physicians place importance on preventing more attacks, with most people living with HAE preferring a prophylactic treatment that is effective in reducing the number of attacks rather than a treatment that offers less frequent administration,” Jonathan Bernstein, MD, at the University of Cincinnati College of Medicine, said in a press release. 

“A treatment that may offer less frequent administration may seem appealing, but these findings suggest that many people living with HAE live with daily fear, stress, and anxiety related to having an attack,” he added.

HAE is mostly caused by a deficiency in C1 inhibitor, a protein in the blood that helps control swelling, its hallmark symptom. CSL makes two HAE therapies, Haegarda and Berinert, both of which are solutions of the missing C1 inhibitor protein. 

Berinert is administered intravenously (into-the-vein) during acute symptomatic attacks, while Haegarda aims to prevent attacks by regular subcutaneous (under-the-skin) injection.

According to the survey, 43% of HAE patients say the disease has a major negative impact on every aspect of their lives, whereas 9% of physicians say the same about their patients. Furthermore, 68% of patients think living with HAE has a major or moderate impact on their work or studies.

“HAE affects my life daily; with the concerns and stress of not only maintaining my own health as a patient but also that of my children, both of whom also have HAE,” said Cheryl French.

Other questions regarding the impact of HAE on lifestyle found 76% of HAE patients worry about having another attack every day, and over 80% consider recovering from an attack to be disruptive to their quality of life.

A total of 70% of people with HAE rate their attacks as severe or very severe, and only 35% think their HAE is very well controlled.

Concerning treatment, more than 95% of patients say they will take whatever measures are necessary to avoid attacks, and 82% of physicians agree. Moreover, 97% of people with HAE, and 99% of physicians agree that preventing attacks is the best way to manage HAE. 

These findings demonstrate that HAE patients and their physicians are generally aligned when it comes to treatment choices. Still, the results also found that patients and clinicians may benefit from a better understanding of mutual priorities in selecting a therapy, the company said. 

“These substantial burdens should be important points of consideration when discussing and developing treatment plans,” Bernstein said.