Získaná trombotická trombocytopenická purpura

Abstract

Thrombotic thrombocytopenic purpura (TTP) is a rare, life-threatening disease belonging to the group of thrombotic microangiopathies (TMA). It is characterised by the presence of microangiopathic haemolytic anaemia, severe thrombocytopenia and ischaemic end-organ damage. The underlying cause of the disease is a severe deficiency of von Willebrand factor cleaving metalloprotease. The enzyme deficiency causes the accumulation of large multimers of vWF to which platelets actively bind, with subsequent formation of microthrombi in the microcirculation. The most important test for the diagnosis of TTP is reduced ADAMTS13 enzyme activity below 10 % (0.1 IU/ml). Treatment of TTP should be initiated as early as possible when TTP is suspected, often before the ADAMTS13 activity results are known. The current standard of care for acute attacks of acquired TTP includes therapeutic plasma exchange, immunosuppression and caplacizumab. A rapid diagnosis of TTP, including ADAMTS13 activity testing, and early initiation of comprehensive treatment are critical to treatment success. Long-term monitoring of disease activity, including monitoring of ADAMTS13 activity, is also the necessary part of the treatment

Key words: Thrombotic thrombocytopenic purpura, ADAMTS13, therapeutic plasma exchange, steroids, rituximab, caplacizumab.