open access

Vol 8, No 1 (2017)
Review paper
Published online: 2017-05-02
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Practical aspects of diagnosis and treatment of thrombotic thrombocytopenic purpura

Magdalena Górska-Kosicka1, Jerzy Windyga12
DOI: 10.5603/Hem.2017.0002
·
Hematologia 2017;8(1):12-19.
Affiliations
  1. Poradnia Leczenia Żywieniowego, Instytut Hematologii i Transfuzjologii, Warszawa, Poland
  2. Zakład Hemostazy i Chor ób Metabolicznych, Instytut Hematologii i Transfuzjologii, Warszawa, Poland

open access

Vol 8, No 1 (2017)
REVIEW ARTICLES
Published online: 2017-05-02

Abstract

Thrombotic thrombocytopenic purpura (TTP) is characterized by a triad of symptoms: thrombocytopenia, hemolytic anemia and organ dysfunction due to disturbed microcirculation. TTP is caused by severe deficiency of a plasma metalloprotease, ADAMTS13 (a disintegrin and metalloprotease with thrombospondin type 1 motifs 13) either due to congenital defects in the ADAMTS13 gene (congenital TTP) or development of autoantiobodies against ADAMTS13 (acquired TTP). Organ dysfunction is life threatening and may lead to death unless patients are immediately applied a therapy of plasma exchange (PEX). Initial diagnosis of TTP is based on clinical symptoms of ischemic organ injury, thrombocytopenia and hemolytic anemia with negative Coombs test and schistocytes in blood smear. Suspected TTP requires collection of pretreatment samples for measure­ment of ADAMTS3 activity and level of anti-ADAMTS13 autoantibodies. PEX should be initiated as soon as possible, even before the results of ADAMTS13 activity and anti-ADAMTS13 autoan­tibodies become available. ADAMTS13 activity below 5–10% with the presence of ADAMTS13 autoantibodies confirms the diagnosis of acquired TTP and daily PEX in combination with im­munosuppressive treatment (corticosteroids) should be continued. Diagnosis of congenital TTP is confirmed by low ADAMTS13 activity in the absence of autoantibodies to ADAMTS13. Congenital TTP is usually treated with infusions of fresh frozen plasma to substitute the missing enzyme.

Abstract

Thrombotic thrombocytopenic purpura (TTP) is characterized by a triad of symptoms: thrombocytopenia, hemolytic anemia and organ dysfunction due to disturbed microcirculation. TTP is caused by severe deficiency of a plasma metalloprotease, ADAMTS13 (a disintegrin and metalloprotease with thrombospondin type 1 motifs 13) either due to congenital defects in the ADAMTS13 gene (congenital TTP) or development of autoantiobodies against ADAMTS13 (acquired TTP). Organ dysfunction is life threatening and may lead to death unless patients are immediately applied a therapy of plasma exchange (PEX). Initial diagnosis of TTP is based on clinical symptoms of ischemic organ injury, thrombocytopenia and hemolytic anemia with negative Coombs test and schistocytes in blood smear. Suspected TTP requires collection of pretreatment samples for measure­ment of ADAMTS3 activity and level of anti-ADAMTS13 autoantibodies. PEX should be initiated as soon as possible, even before the results of ADAMTS13 activity and anti-ADAMTS13 autoan­tibodies become available. ADAMTS13 activity below 5–10% with the presence of ADAMTS13 autoantibodies confirms the diagnosis of acquired TTP and daily PEX in combination with im­munosuppressive treatment (corticosteroids) should be continued. Diagnosis of congenital TTP is confirmed by low ADAMTS13 activity in the absence of autoantibodies to ADAMTS13. Congenital TTP is usually treated with infusions of fresh frozen plasma to substitute the missing enzyme.

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Keywords

thrombotic thrombocytopenic purpura, ADAMTS13, Upshaw-Schulman syndrome, schistocytes, plasma exchange, immunosuppression

About this article
Title

Practical aspects of diagnosis and treatment of thrombotic thrombocytopenic purpura

Journal

Hematology in Clinical Practice

Issue

Vol 8, No 1 (2017)

Article type

Review paper

Pages

12-19

Published online

2017-05-02

DOI

10.5603/Hem.2017.0002

Bibliographic record

Hematologia 2017;8(1):12-19.

Keywords

thrombotic thrombocytopenic purpura
ADAMTS13
Upshaw-Schulman syndrome
schistocytes
plasma exchange
immunosuppression

Authors

Magdalena Górska-Kosicka
Jerzy Windyga

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