Short-term Use of Ruconest Can Prevent HAE Attacks Triggered by Medical or Dental Work, Study Reports
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.
The study, “Recombinant Human C1 Esterase Inhibitor as Short-Term Prophylaxis in Patients With Hereditary Angioedema,” was published in The Journal of Allergy and Clinical Immunology: In Practice.
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.
Such acute attacks are generally unpredictable, but some triggers are known, like dental or medical procedures, other causes of trauma, and stress.
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.
For the six cases in which an attack occurred between day 2 and day 7, Ruconest was given 60 minutes or less (three cases), 120 minutes (one case), and 280 minutes (one case) prior to the procedure; in one case, the time of treatment administration was not reported.
When patients on long-term prophylaxis were excluded from the analysis (19 procedures), 96.1% of the cases treated with Ruconest did not result in any attack within the first two days post-procedure, and and 88.2% within seven days.
These data support “a high rate of success with rhC1-INH [Ruconest] as prophylaxis, irrespective of concomitant long-term prophylaxis use,” the researchers wrote.
In all cases, preventive use Ruconest was safe and well tolerated, with no adverse events reported.
The findings suggest that short-term prophylaxis with the medicine reduces the risk of angioedema attacks, following exposure to potentially triggering events.
Researchers also noted that Ruconest benefits are comparable to those reported form short-term prophylaxis with plasma-derived C1-INH concentrates (marketed as Berinert, Cinryze and Haegarda).
“As suggested by an international guideline, C1-INH concentrate should be administered as close as possible to the time of procedure initiation. On-demand treatment should also be available to manage breakthrough HAE attacks” they added.
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.
Ana Pena, PhD