Short-term use of Ruconest (recombinant human C1 inhibitor) as a preventive treatment reduces the risk of hereditary angioedema (HAE) attacks triggered by medical and dental procedures or by stress, a case series reports.
Hereditary angioedema (HAE) is a rare genetic disease caused by a deficiency in (type 1) or the production of a less-effective (type 2) C1 esterase inhibitor (C1-INH), or C1 inhibitor. Lack of this protein activates a chain of reactions in the blood that increases the permeability of blood vessels.
People with HAE frequently experience sudden episodes of swelling in the deeper layers of the skin, usually not accompanied by hives or rash, that are often painful.
To avoid such flares, short-term preventive treatment (prophylaxis) with C1-INH concentrates is recommended before surgery or dental procedures.
Ruconest is a recombinant human C1-INH, or rhC1-INH, marketed by Pharming Group. It is given as infusions into the vein and approved in the U.S. and European Union to treat HAE attacks in adults and adolescents.
The treatment is also known to be effective and well tolerated for long-term prophylaxis in people with frequent attacks. But little is known to date about its benefits as shorter term preventive therapy.
An international team of researchers, sponsored by Pharming Group, collected and analyzed data from 51 HAE patients given short-term prophylaxis with Ruconest.
These 51 patients, from the U.S. and Europe (median age of 44), were treated with Ruconest shortly before a medical (surgery, endoscopy) or dental procedure (e.g., tooth extraction, oral surgery, and cutting of soft tissues), or a stressful life event.
Data from 70 procedures and the attacks observed over one week following each procedure or event were analyzed. A 16-person subset of these same patients served as a control group; 26 procedures they underwent at a different occasion were done without any prophylactic treatment (long- or short-term). Most patients had type 1 HAE (92.2%), and the whole group had a median of 14 attacks per year.
In most cases, (68.6%) Ruconest was given 10 to 65 minutes prior to each procedure. Doses ranged from 2,100 to 4,200 IU.
Within the first seven days, 88.6% of those receiving short-term prophylaxis were free of attacks, compared with 19.2% in the control group.
Although children and adolescents were not included in the current study, researchers still recommend short-term prophylaxis for young patients about to undertake potentially threatening procedures.