Study Shows Efficacy of Treatment for Preventing Hereditary Angioedema Attacks

About HAE

Hereditary angioedema (HAE) is a rare disease that is caused by the deficiency or reduction of a protein called C1-INH in the body. When this protein is not present, the permeability of blood vessels is increased. This results in episodes of swelling, hives, rashes, and pain.

HAE flares can come with no warning, as a completely unpredictable event. That said, there are some triggers known to cause attacks. These include stress and trauma as well as medical procedures or dental care.

Ruconest

Ruconest is a C1 inhibitor. An international team of researchers conducted an investigation to see whether this medication could be utilized as a preventative therapy for HAE attacks. To examine this effect, they specifically examined situations where events are predictable.

The therapy is administered as an infusion directly into the vein. It has already been approved in both the United States and Europe. Typically, the medication is utilized for long-term prophylaxis. However, not much research has been done on its short-term effects. This is what this study aimed to uncover.

Could Ruconest be used immediately prior to triggering events to eliminate the chance of an HAE attack?

The Study

This study included a total of 51 HAE patients from both the United States and Europe. Their median age was 44 and their median number of HAE attacks experienced per year was 14. 92.2% of the participants were diagnosed with type 1 HAE. Although children were not included in this study, the team of researchers still believe the same results would be found with this population and recommend the same care.

The patients in this study were each about to have a medical procedure, dental procedure, or undergo a stressful event. There were 70 procedures occurring in total across the participants. The patients were observed for 7 days following their procedure or event.
16 of the patients, accounting for 26 of the procedures, were a part of the control group. These individuals were not given short-term or long-term prophylactic treatment. For the remainder of the patients, Ruconest was administered soon before their procedure (for most, 10-65 minutes before).
The dose of the medication varied per patient (between 2,100 to 4,200 IU).

In the first 7 days, 88.6% of patients in the treatment group experienced no attacks. Of those in the control group, only 19.2% were attack-free.

No adverse events were documented from this investigation, with Ruconest proving safe and tolerable for patients. Ultimately, these findings show that Ruconest was able to reduce the risk of an attack after a triggering event such as a medical procedure. Researchers recommend, based on this study, that the medication should be administered to the patient as close as possible to the moment that they will be undergoing the procedure or event.

Ultimately, the results of this study are exciting because it means that HAE patients may be able to more effectively prevent attacks, significantly improving their quality of life.

The benefits of Ruconest were found to be similar to Berinert, Cinryze, and Haegarda.

You can read more about this study and its findings here.

You can read the full study in The Journal of Allergy and Clinical Immunology: In Practice.


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