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Recombinant ADAMT S-13 as a new alternative for the patients with congenital thrombocytopenic purpura

Year: 2024

Type: article

Abstract: Congenital thrombocytopenic purpura (cTTP), also known as Upshaw–Schulman syndrome is a rare disease, caused by ADAMTS13 deficiency. This condition may result in various complications, including haemolytic anaemia, renal dysfunction or thrombotic events. To this very day, the most common treatment of this condition is transfusion of fresh frozen plasma. Recently, recombinant ADAMTS13 (rADAMTS13) has been introduced, representing a revolutionary advancement in the treatment of cTTP, and giving patients new, more comfortable alternative. We present here a case report of a patient with congenital thrombocytopenic purpura, who after 30 years of being treated with FFP transfusions and developing complications from both the disease and its treatment, was successfully transitioned to rADAMTS13 therapy.

Source: Oncoreview

DOI: 10.24292/01.or.143301224

Authors: Izabela Filar, Michał Witkowski, Wiktoria Ryżewska, Tadeusz Robak

Institutions: Wojskowy Instytut Medycyny Lotniczej, Copernicus Memorial Hospital, Medical University of Lodz

Language: English

Topic: Complement system in diseases

Subfield: Immunology

Field: Immunology and Microbiology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: diamond

OpenAlex ID: W4408911811

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