Active substance

Results for the substance radamts13

2 results

About this active substance

Recombinant ADAMTS13 is an enzyme replacement therapy used in patients with congenital thrombotic thrombocytopenic purpura, a rare disease in which the ADAMTS13 enzyme is missing or works insufficiently. Deficiency leads to small blood clots in vessels, with risk of low platelets, anaemia, neurological problems, kidney injury and other complications.

It is given by intravenous injection under the supervision of a doctor experienced in haematological disorders. It may be used preventively at regular intervals or to treat an acute episode, depending on the patient’s condition. The dose is calculated by body weight and may be adjusted according to response, laboratory values and special situations such as infection or procedures.

Side effects may include headache, nausea, diarrhoea, dizziness, vomiting, abdominal pain, migraine or respiratory infections. Allergic reactions may occur, including rash, hives, tight throat, chest pain, breathing difficulty or fainting. There is also a risk of antibody formation, which may reduce the effectiveness of treatment.

Contact a doctor urgently if unusual bruising or bleeding, confusion, one-sided weakness, chest pain, shortness of breath, severe rash, fever, worsening tiredness or signs of allergic reaction occur. Keeping the treatment schedule and blood-test appointments is essential. Tell the doctor about pregnancy, breastfeeding, infections and any reactions to plasma-derived products.