C1inhibitorsersättning

C1 inhibitor replacement therapy, often referred to as C1-INH replacement or simply augmentation therapy, is a treatment used for hereditary angioedema (HAE). HAE is a rare genetic disorder characterized by recurrent episodes of swelling in various parts of the body. This swelling is caused by a deficiency or dysfunction of the C1 inhibitor protein, a key component of the complement system, which helps regulate inflammation. When C1 inhibitor is not functioning correctly, it leads to the overproduction of bradykinin, a substance that increases blood vessel permeability and results in swelling.

C1 inhibitor replacement therapy aims to restore normal levels of functional C1 inhibitor in the blood. It is typically administered intravenously and can be used in two main ways: acutely to treat ongoing HAE attacks and prevent them from worsening, or prophylactically to reduce the frequency and severity of attacks. The therapy involves infusing purified human plasma-derived C1 inhibitor concentrate. Patients with HAE are usually educated on how to self-administer this treatment at home, allowing for prompt intervention during an attack. While effective, C1 inhibitor replacement therapy carries some risks, including allergic reactions and the potential transmission of infectious agents, though these risks are minimized through rigorous screening and purification processes. The development of this therapy has significantly improved the quality of life for individuals with HAE by providing a way to manage and prevent debilitating swelling episodes.