The study examined and compared treatment outcomes for human C1 inhibitors (plasmatic and recombinant) and for icatibant, a registered bradykinin inhibitor. The results support additional clinical investigations to explore the use of C1 inhibitors (pdC1INH or rhC1INH) as first-line attack resolution therapy. The study was led by Professor Dr Marcus Maurer and Professor Dr Markus Magerl at Charité – Universitätsmedizin Berlin, Germany.
Hereditary angioedema (HAE) is a rare genetic disorder. It is characterised by spontaneous and recurrent episodes of swelling (edema attacks) of the skin in different parts of the body, as well as in the airways and internal organs.
- Seven patients with HAE who had experienced more than one attack every six weeks in the previous 12 months and had been treated with self-administered on-demand therapy were included in the study.
- The median number of attacks among the seven patients in the previous 12 months was 25, ranging from 13 to 43 attacks.
- During the study period, there were 69 attacks in total across all seven patients, 67 of which were treated.
- Attacks were initially treated with either Berinert® (n=5) or Cinryze® (n=17), both plasma-derived C1 inhibitors (pdC1INH), or Firazyr® (n=25), a small molecule bradykinin inhibitor (icatibant), or Ruconest® (n=20), a recombinant human C1 inhibitor (rhC1INH).
- 14 (21%) attacks were treated with a second dose or a second treatment; 0 (0%), 1 (6%), 11 (44%), and 2 (10%) attacks were initially treated with Berinert®, Cinryze®, Firazyr®, and Ruconest®, respectively. All rebound attacks after initial treatment with Cinryze® or Firazyr® were treated with the recommended dose, whereas all rebound attacks after initial treatment with Ruconest® happened after initial underdosing (2100U).
- All attacks that required subsequent therapy and were treated with a C1 inhibitor (either pdC1INH or rhC1INH) resolved without further treatment. In contrast, some attacks re-treated with icatibant did not resolve and required one or more further subsequent treatment. When C1 was used for subsequent therapy, the therapy was effective. When icatibant was used for subsequent therapy, the therapy was not always effective.
- Three attacks showed no improvement (1 treated with Cinryze® 1000 units, 2 treated with Firazyr®). One attack required the intervention of emergency medical staff (treated with Cinryze® 1000 units).
Additional data regarding the reasons for drug selection and subjective observations on the performance of the drugs in each attack are being analysed and the full results of the study will be published in due course.
About HAE
Hereditary Angioedema (HAE) is a rare genetic disorder. It is characterised by spontaneous and recurrent episodes of swelling (edema attacks) of the skin in different parts of the body, as well as in the airways and internal organs. Edema of the skin usually affects the extremities, the face, and the genitals. Patients suffering from this kind of edema often withdraw from their social lives because of the disfiguration, discomfort and pain these symptoms may cause. Almost all HAE patients suffer from bouts of severe abdominal pain, nausea, vomiting and diarrhea caused by swelling of the intestinal wall.
Edema of the throat, nose or tongue can be particularly dangerous as this can lead to obstruction of the airway passages and be potentially life threatening. Although there is currently no known cure for HAE, it is possible to treat the symptoms associated with edema attacks. HAE affects about 1 in 10,000 to 1 in 50,000 people. Worldwide experts believe that a lot of patients are still seeking the right diagnosis: although HAE is (in principle) easy to diagnose, it is frequently identified very late or not discovered at all. The reason HAE is often misdiagnosed is because the symptoms are similar to those of many other common conditions such as allergies or appendicitis. By the time it is diagnosed correctly, the patient has often been through a long-lasting ordeal.
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