Thrombotic thrombocytopenic purpura: recognition and management

Int J Hematol. 2010 Jan;91(1):36-45. doi: 10.1007/s12185-009-0478-z.

Abstract

Thrombotic thrombocytopenic purpura is a life-threatening multisystem disorder that represents both a diagnostic and a management challenge to clinicians. Early recognition of the condition coupled with rapid institution of plasma exchange has led to a dramatic improvement in prognosis. Studies performed over the past decade have elucidated the predominant pathophysiology, stemming from a deficiency of ADAMTS13, that accounts for the widespread microvascular deposition of platelet-von Willebrand factor in many sites, including the brain, kidney, and mesenteric vessels. However, in light of the mortality rate of 10-20%, much work remains to be done to translate advances in our understanding of pathophysiology into clinical practice. Improvements in medical management using immunosuppressive and other drugs are being actively explored in clinical trials. Agents that target ADAMTS13 autoantibody production by B-cells, such as anti-CD20 monoclonal antibodies, have the potential to shorten the duration of plasma exchange treatment, reduce relapses, and transform the management of this once enigmatic disorder.

Publication types

  • Review

MeSH terms

  • ADAM Proteins / immunology
  • ADAMTS13 Protein
  • Autoantibodies / blood
  • Humans
  • Immunosuppressive Agents / therapeutic use*
  • Plasma Exchange*
  • Purpura, Thrombotic Thrombocytopenic / diagnosis*
  • Purpura, Thrombotic Thrombocytopenic / drug therapy*
  • Purpura, Thrombotic Thrombocytopenic / immunology

Substances

  • Autoantibodies
  • Immunosuppressive Agents
  • ADAM Proteins
  • ADAMTS13 Protein
  • ADAMTS13 protein, human