printInformation about HAE, treatment options and the drugs used to treat HAE is now available on various internet sites. For example, two consensus documents about the diagnosis and treatment of HAE prepared by international working groups and published in scientific journals are available free of charge on the internet.
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Therapeutical overview
An overview of treatment options
It is our aim to provide you with an overview and, in consultation with an international group of experts, keep it up to date.
We stress that these are not treatment recommendations. We are simply listing the treatment options that are currently available or in development.
Selection and adaptation of a suitable treatment for an individual patient should always and only be undertaken by a doctor who is experienced in the treatment of hereditary angioedema.
To this end, this website contains a list of the HAE centres known to us with all available contact details. This list is updated on an ongoing basis. It can be used to find your nearest HAE centre quickly, e.g. if you move house or travel abroad.
Acute treatment
The aim of acute treatment is to halt the progression of the edema and alleviate the symptoms. This applies particularly to episodes affecting the larynx, which can cause death by suffocation if left untreated.
The recommended options for acute treatment vary from country to country due to the fact that several of the drugs are not licensed in all countries. In these cases acute treatment may be limited to more unspecific drugs such as androgens, tranexamic acid or even just pain killers.
In several European countries, most specific acute treatment currently consists of C1-INH concentrate if available, which can compensate for the C1-INH deficiency directly. If no concentrate is available, fresh frozen blood plasma, which also contains C1-INH, can be used as an alternative. However, as this also exposes the body to new complement factors or bradykinin precursors, which can restart the reaction process, existing swelling may even get worse.
Both C1-INH concentrate and blood plasma usually have to be administered intravenously by a doctor or in hospital. Therefore, patients are often very restricted in their mobility, particularly if they have frequent episodes. Acute treatment of an episode sometimes requires patients to remain in hospital for several days.
The recommended options for acute treatment vary from country to country due to the fact that several of the drugs are not licensed in all countries. In these cases acute treatment may be limited to more unspecific drugs such as androgens, tranexamic acid or even just pain killers.
In several European countries, most specific acute treatment currently consists of C1-INH concentrate if available, which can compensate for the C1-INH deficiency directly. If no concentrate is available, fresh frozen blood plasma, which also contains C1-INH, can be used as an alternative. However, as this also exposes the body to new complement factors or bradykinin precursors, which can restart the reaction process, existing swelling may even get worse.
Both C1-INH concentrate and blood plasma usually have to be administered intravenously by a doctor or in hospital. Therefore, patients are often very restricted in their mobility, particularly if they have frequent episodes. Acute treatment of an episode sometimes requires patients to remain in hospital for several days.
Long-term prophylaxis
Long-term prophylaxis is given to patients whose quality of life is clearly reduced by the disease. These are usually patients who have very frequent episodes or a high risk of developing laryngeal edema.
Long-term prophylaxis consists mainly of attenuated androgens, synthetically produced derivatives of the male sex hormone testosterone. They increase production of C1-INH through an as yet unknown mechanism and have proved useful in reducing the frequency or severity of attacks. A variety of dose dependent side effects is associated with the use of androgens. There have been reports of cases of hepatocellular adenoma developing in patients on longterm prophylaxis with danazol.
As an alternative to androgens, the fibrinolysis inhibitor tranexamic acid is used in some countries. Although it is not as effective as an androgen and potential side effects make its use controversial (you may wish to compare consensus documents).
Long-term prophylaxis consists mainly of attenuated androgens, synthetically produced derivatives of the male sex hormone testosterone. They increase production of C1-INH through an as yet unknown mechanism and have proved useful in reducing the frequency or severity of attacks. A variety of dose dependent side effects is associated with the use of androgens. There have been reports of cases of hepatocellular adenoma developing in patients on longterm prophylaxis with danazol.
As an alternative to androgens, the fibrinolysis inhibitor tranexamic acid is used in some countries. Although it is not as effective as an androgen and potential side effects make its use controversial (you may wish to compare consensus documents).
Short-term prophylaxis
Surgical procedures or dental treatment can lead to episodes of swelling. In order to avoid this, HAE patients usually receive short-term prophylaxis beforehand. Where registered, C1-INH concentrate is used for this purpose and is administered 12-24 hours before the procedure. In other European countries, treatment with high-dose androgens for five to seven days is used.
Future treatment options (under development)
Current treatment of hereditary angioedema is inadequate in many countries and therefore new treatment options are under clinical development: such as DX-88 a genetically engineered (recombinant) kallikrein inhibitor and Icatibant, a bradykinin receptor angatonist for subcutaneous injection, as well as recombinant (Rhucin®) and plasma derived C1-INH concentrate (Cynrize®), that have to be administered intravenously.
Short summary (tabular overview)
| Acute treatment | Long-Term Prophylaxis | Short-Term Prophylaxis |
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