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Thrombotic Thrombocytopenic Purpura, Atypical Hemolytic Uremic Syndrome, and Spectrum of Thrombotic Microangiopathy

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Pediatric Nephrology

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Abstract

Thrombotic thrombocytopenic purpura (TTP) and hemolytic uremic syndrome (HUS) are thrombotic microangiopathies (TMAs) which are characterized by microangiopathic hemolytic anemia (MAHA) and thrombocytopenia and show significant overlap in clinical presentation but are distinct in their underlying pathology.

TTP is caused by an inherited or acquired deficiency in a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 (ADAMTS13). ADAMTS13 cleaves von Willebrand factor (VWF) when released from endothelial cells, thus preventing the accumulation of prothrombotic ultra-large VWF (ULFWF) multimers. By difference, hemolytic uremic syndrome (HUS) is caused by infections with Shiga toxin (Stx)-producing Escherichia coli (E. coli; STEC HUS). And in the atypical form of HUS (aHUS), the endothelial injury is caused by complement dysregulation due to genetic (gain- or loss-of-function mutations) or autoimmune (inhibiting autoantibodies) causes. Recently, transient complement dysregulation has also been recognized in the absence of mutations and autoantibodies but rather as a consequence of infections, autoimmune diseases, drugs, mechanical endothelial injury, etc., thus defining a spectrum of secondary TMAs.

The advent of high precision medications which allow for the safe and efficient treatment of aHUS and TTP has fundamentally changed patient outcomes and quality of life.

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Authors and Affiliations

  1. Division of Nephrology, The Hospital for Sick Children, Toronto, ON, Canada

    Magdalena Riedl Khursigara, Mathieu Lemaire & Christoph Licht

  2. Division of Nephrology and Hypertension, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, USA

    Stefanie Benoit

  3. Department of Pediatrics, College of Medicine, University of Cincinnati, Cincinnati, OH, USA

    Stefanie Benoit

  4. Division of Hematology, University of Toronto, Toronto, ON, Canada

    Christopher J. Patriquin

  5. Division of Medical Oncology and Hematology, University Health Network, Toronto, ON, Canada

    Christopher J. Patriquin

  6. Department of Pediatrics, University of Toronto, Toronto, ON, Canada

    Mathieu Lemaire & Christoph Licht

Authors
  1. Magdalena Riedl Khursigara
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  2. Stefanie Benoit
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  3. Christopher J. Patriquin
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  4. Mathieu Lemaire
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  5. Christoph Licht
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Corresponding author

Correspondence to Christoph Licht .

Editor information

Editors and Affiliations

  1. Division of Nephrology, Bambino Gesù Children’s Hospital –, ROMA, Roma, Italy

    Francesco Emma

  2. Division of Nephrology and Hypertension The Heart Institute, University of Cincinnati Cincinnati Children’s Hospital Medical C, Cincinnati, OH, USA

    Stuart Goldstein

  3. Div of Nephrology, Dept of Pediatrics, All India Institute of Medical Sciences, New Delhi, Delhi, India

    Arvind Bagga

  4. Division of Pediatric Nephrology, UPMC Children’s Hospital of Pittsburgh, Pittsburgh, PA, USA

    Carlton M. Bates

  5. Renal Unit, Graet Ormond Street Hospital, London, UK

    Rukshana Shroff

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Riedl Khursigara, M., Benoit, S., Patriquin, C.J., Lemaire, M., Licht, C. (2022). Thrombotic Thrombocytopenic Purpura, Atypical Hemolytic Uremic Syndrome, and Spectrum of Thrombotic Microangiopathy. In: Emma, F., Goldstein, S., Bagga, A., Bates, C.M., Shroff, R. (eds) Pediatric Nephrology. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-642-27843-3_43-2

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  • DOI: https://doi.org/10.1007/978-3-642-27843-3_43-2

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  • Publisher Name: Springer, Berlin, Heidelberg

  • Print ISBN: 978-3-642-27843-3

  • Online ISBN: 978-3-642-27843-3

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  1. Latest

    Thrombotic Thrombocytopenic Purpura, Atypical Hemolytic Uremic Syndrome, and Spectrum of Thrombotic Microangiopathy
    Published:
    21 July 2022

    DOI: https://doi.org/10.1007/978-3-642-27843-3_43-2

  2. Original

    Published:
    10 February 2015

    DOI: https://doi.org/10.1007/978-3-642-27843-3_43-1

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