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TTP and aHUS: new insights.

Yoshitaka Miyakawa1

  • 1Department of General Internal Medicine, Center for Thrombosis and Hemostasis, Saitama Medical University Hospital.

[Rinsho Ketsueki] the Japanese Journal of Clinical Hematology
|June 9, 2017
PubMed
Summary
This summary is machine-generated.

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Rilzabrutinib for persistent or chronic immune thrombocytopenia (ITP): a Bruton tyrosine kinase inhibitor that can target multiple pathophysiological pathways.

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Efgartigimod as a treatment for adults with primary immune thrombocytopenia: a plain language summary of the ADVANCE IV study.

Therapeutic advances in hematology·2026
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Safety and Effectiveness of Sutimlimab in Cold Agglutinin Disease: A Real-World International Experience.

American journal of hematology·2026
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Efficacy and Safety of Subcutaneous Efgartigimod PH20 in Adults With Primary Immune Thrombocytopenia (ADVANCE SC): A Multicenter, Randomized, Double-Blinded, Placebo-Controlled, Phase 3 Trial.

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Warm Autoimmune Haemolytic Anaemia Management in Asia-Pacific: A Delphi Panel Expert Consensus and Systematic Literature Review.

EJHaem·2025

Thrombotic thrombocytopenic purpura (TTP) and atypical hemolytic uremic syndrome (aHUS) are rare intractable diseases. This article provides an updated overview for hematology/oncology professionals, highlighting their shared thrombotic microangiopathy pathophysiology.

Area of Science:

  • Hematology
  • Oncology
  • Rare Diseases

Background:

  • Thrombotic thrombocytopenic purpura (TTP) and atypical hemolytic uremic syndrome (aHUS) are recognized as intractable diseases in Japan since 2015.
  • Both TTP and aHUS share common pathophysiological mechanisms, notably thrombotic microangiopathy.
  • There is a notable lack of awareness regarding TTP and aHUS among hematology/oncology professionals.

Purpose of the Study:

  • To provide an updated overview of TTP and aHUS.
  • To increase awareness and understanding of these rare hematological disorders among medical professionals.
  • To discuss the shared pathophysiology of TTP and aHUS.

Main Methods:

  • Review of current literature and clinical practice guidelines for TTP and aHUS.
Keywords:
EculizumabRituximabTMATTPaHUS

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  • Comparative analysis of the pathophysiology of TTP and aHUS.
  • Synthesis of information to create an accessible overview for healthcare providers.

    • Main Results:

    • TTP and aHUS are designated intractable diseases, with updated clinical guidelines available.
    • Thrombotic microangiopathy is a key shared pathophysiological feature.
    • A knowledge gap exists among hematology/oncology professionals regarding these conditions.

    Conclusions:

    • Increased awareness and understanding of TTP and aHUS are crucial for timely diagnosis and management.
    • The shared pathophysiology underscores the need for integrated approaches in studying and treating these diseases.
    • Dissemination of updated information and guidelines is essential for improving patient outcomes in TTP and aHUS.