Successful Treatment With Bortezomib for Refractory and Complicated Acquired Thrombotic Thrombocytopenic Purpura in an Adolescent Girl

J Pediatr Hematol Oncol. 2021 May 1;43(4):e587-e591. doi: 10.1097/MPH.0000000000002026.


Thrombotic thrombocytopenic purpura (TTP) is a rare, dangerous, life-threatening disease characterized by microangiopathic hemolytic anemia and thrombocytopenia, along with organ dysfunction due to microangiopathy-related ischemia. Plasma exchange and steroids are used for initial treatment, and rituximab is often used in refractive patients. Caplacizumab, cyclophosphamide, and splenectomy are among other treatment options. It has been reported that bortezomib, a proteasome inhibitor, can be used in the management of refractory acquired TTP. Herein, we present a 16-year-old female patient who was monitored for acquired TTP and treated with high-dose steroids, plasma exchange, rituximab, cyclophosphamide, and N-acetylcysteine but developed renal, cardiac, gastrointestinal, and neurologic complications. The girl was then successfully treated with bortezomib, and she has been monitored in remission for 6 months. We consider that bortezomib is a beneficial treatment, especially in patients with refractory TTP.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Bortezomib / therapeutic use*
  • Female
  • Humans
  • Plasma Exchange
  • Proteasome Inhibitors / therapeutic use*
  • Purpura, Thrombotic Thrombocytopenic / complications
  • Purpura, Thrombotic Thrombocytopenic / drug therapy*
  • Purpura, Thrombotic Thrombocytopenic / therapy
  • Treatment Outcome


  • Proteasome Inhibitors
  • Bortezomib

Supplementary concepts

  • Thrombotic thrombocytopenic purpura, acquired