title: Bortezomib for chronic relapsing thrombotic thrombocytopenic purpura: a case report.
abstract: BACKGROUND
  Acquired thrombotic thrombocytopenic purpura (TTP) is an autoimmune disorder characterized by a severe deficiency of ADAMTS13 activity. Although therapeutic plasma exchange (PLEX) is the standard of care, 30% to 50% patients develop exacerbation or relapse, requiring immunomodulatory agents. Of these agents, glucocorticoids, rituximab, and cyclosporine A are the most frequently used.
  
  
  METHODS
  We report a case of chronic relapsing TTP in a patient who had eight relapses over a 14-year period. After her seventh relapse, the patient demonstrated only partial response to glucocorticoids, two courses of rituximab, and cyclophosphamide. The eighth relapse occurred 58 days after her last PLEX and subsequent to this she received a course of bortezomib (Velcade, Millennium Pharmaceuticals, Inc.). After treatment with bortezomib the patient demonstrated a complete response with a progressive increase in ADAMTS13 activity from less than 5% to 22% accompanied by undetectable inhibitor, and she has remained PLEX free for more than 169 days.
  
  
  CONCLUSIONS
  Bortezomib may serve as an adjunct treatment in patients with acquired TTP who exhibit an incomplete response or are refractory to conventional management.
authors.affiliation: Department of Pathology, Division of Transfusion Medicine and Hemostasis, University of Texas Southwestern Medical Center, Dallas, Texas.
authors.forename: Sean
authors.initials: S
authors.lastname: Yates
authors.forename: Karen
authors.initials: K
authors.lastname: Matevosyan
authors.forename: Cynthia
authors.initials: C
authors.lastname: Rutherford
authors.forename: Yu-Min
authors.initials: YM
authors.lastname: Shen
authors.forename: Ravi
authors.initials: R
authors.lastname: Sarode
mesh_terms: D051722:ADAM Proteins / Q000097:blood / Q000276:immunology; D000071120:ADAMTS13 Protein; D058846:Antibodies, Monoclonal, Murine-Derived / Q000627:therapeutic use; D001897:Boronic Acids / Q000009:adverse effects / Q000627:therapeutic use*; D000069286:Bortezomib; D003520:Cyclophosphamide / Q000627:therapeutic use; D004351:Drug Resistance; D005260:Female; D005938:Glucocorticoids / Q000627:therapeutic use; D006801:Humans; D016867:Immunocompromised Host; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use*; D008875:Middle Aged; D011697:Purpura, Thrombotic Thrombocytopenic / Q000188:drug therapy* / Q000628:therapy; D011719:Pyrazines / Q000009:adverse effects / Q000627:therapeutic use*; D012008:Recurrence; D012074:Remission Induction; D000069283:Rituximab; D013203:Staphylococcal Infections / Q000209:etiology
chemical_list: D058846:Antibodies, Monoclonal, Murine-Derived; D001897:Boronic Acids; D005938:Glucocorticoids; D007166:Immunosuppressive Agents; D011719:Pyrazines; D000069283:Rituximab; D000069286:Bortezomib; D003520:Cyclophosphamide; D051722:ADAM Proteins; D000071120:ADAMTS13 Protein; C099604:ADAMTS13 protein, human