Relapsing or refractory idiopathic thrombotic thrombocytopenic purpura-hemolytic uremic syndrome: the role of rituximab

Transfusion. 2010 Dec;50(12):2753-60. doi: 10.1111/j.1537-2995.2010.02763.x.


Idiopathic thrombotic thrombocytopenic purpura-hemolytic uremic syndrome (TTP-HUS) is a rare disease responsive to treatment with plasma exchange (PE) but with a high percentage of relapse or refractory patients. A severe deficiency of ADAMTS-13 (<5% of normal activity), congenital or caused by an autoantibody, may be specific for TTP and it has been proposed that severe ADAMTS-13 deficiency now defines TTP. B cells play a key role in both the development and the perpetuation of autoimmunity, suggesting that B-cell depletion could be a valuable treatment approach for patients with idiopathic TTP-HUS. This review of the literature focuses on the role of rituximab, a chimeric monoclonal antibody directed against CD20 antigen expressed by B lymphocytes, in patients with relapsing or refractory TTP-HUS with or without ADAMTS-13 deficiency, suggesting that rituximab may produce clinical remission in a significant proportion of patients. Rituximab therapy reduces plasma requirement and avoids complications related to salvage-immunosuppressive therapy. In conclusion, rituximab provides an effective, well-tolerated, and safe treatment option for patients with idiopathic TTP-HUS, thus giving an alternative approach to the current treatment based on PE.

Publication types

  • Evaluation Study
  • Review

MeSH terms

  • ADAM Proteins / antagonists & inhibitors
  • ADAM Proteins / immunology
  • ADAMTS13 Protein
  • Antibodies, Monoclonal / administration & dosage
  • Antibodies, Monoclonal / therapeutic use
  • Antibodies, Monoclonal, Murine-Derived / administration & dosage
  • Antibodies, Monoclonal, Murine-Derived / physiology*
  • Antibodies, Monoclonal, Murine-Derived / therapeutic use*
  • Drug Resistance* / physiology
  • Humans
  • Immunologic Factors / pharmacology
  • Immunologic Factors / therapeutic use
  • Immunotherapy / methods
  • Purpura, Thrombotic Thrombocytopenic / therapy*
  • Recurrence
  • Rituximab


  • Antibodies, Monoclonal
  • Antibodies, Monoclonal, Murine-Derived
  • Immunologic Factors
  • Rituximab
  • ADAM Proteins
  • ADAMTS13 Protein
  • ADAMTS13 protein, human