Severe primary refractory thrombotic thrombocytopenic purpura (TTP) in the post plasma exchange (PEX) and rituximab era

BMJ Case Rep. 2020 Jun 11;13(6):e234091. doi: 10.1136/bcr-2019-234091.

Abstract

Acute acquired thrombotic thrombocytopenic purpura (TTP) requires prompt recognition and initiation of plasma exchange (PEX) therapy and immunosuppression. When PEX fails, mortality nears 100%, making finding an effective treatment crucial. Primary refractory TTP occurs when initial therapies fail or if exacerbations occur during PEX therapy, both signifying the need for treatment intensification to achieve clinical remission. Rituximab helps treat most of the refractory TTP cases, except those that are severely refractory. A paucity of studies guiding severely refractory TTP makes management arbitrary and individualised, highlighting the value of isolated reports. We present an extremely rare case of primary refractory TTP with an insufficient platelet response to numerous types of treatments, including emerging therapies such as caplacizumab, on the background of repeated PEX and immunosuppressive therapies.

Keywords: drugs and medicines; haematology (incl blood transfusion); oncology; thrombotic thrombocytopenic purpura.

Publication types

  • Case Reports

MeSH terms

  • ADAMTS13 Protein / analysis*
  • Dizziness / diagnosis
  • Dizziness / etiology
  • Drug Resistance
  • Fibrinolytic Agents / administration & dosage
  • Humans
  • Immunoglobulins, Intravenous / administration & dosage*
  • Immunosuppressive Agents / administration & dosage
  • Male
  • Middle Aged
  • Mycophenolic Acid / administration & dosage*
  • Neurologic Examination / methods
  • Plasma Exchange / methods*
  • Platelet Count / methods
  • Purpura, Thrombotic Thrombocytopenic* / blood
  • Purpura, Thrombotic Thrombocytopenic* / physiopathology
  • Purpura, Thrombotic Thrombocytopenic* / therapy
  • Rituximab / administration & dosage*
  • Secondary Prevention / methods
  • Severity of Illness Index
  • Single-Domain Antibodies / administration & dosage*
  • Syncope / diagnosis
  • Syncope / etiology
  • Treatment Outcome
  • von Willebrand Factor / antagonists & inhibitors

Substances

  • Fibrinolytic Agents
  • Immunoglobulins, Intravenous
  • Immunosuppressive Agents
  • Single-Domain Antibodies
  • von Willebrand Factor
  • caplacizumab
  • Rituximab
  • ADAMTS13 Protein
  • Mycophenolic Acid

Supplementary concepts

  • Thrombotic thrombocytopenic purpura, acquired