Mycophenolate mofetil therapy for severe immune thrombocytopenia

Br J Haematol. 2015 Nov;171(4):625-30. doi: 10.1111/bjh.13622. Epub 2015 Aug 6.


Severe immune thrombocytopenia purpura (ITP) presents a clinical challenge. Second-line treatment options are variable without a precise protocol. We present 46 severe ITP patients treated with mycophenolate mofetil (MMF), retrospectively identified from three London teaching hospitals. Data was collected on patient demographics, co-morbidities and previous treatment strategies. Our key interest was whether there was a sustained response in platelet count to MMF. Patients included 27 males and 19 females whose ages ranged from 19 to 93 years old (median 52·5 years). Twenty-nine had primary ITP and 17 had secondary ITP, a third of whom had viral-associated disease. The standard dose of MMF was 1 g/day. Twenty-four patients (52%) responded with 15 (33%) achieving a complete response. No active viral-associated ITP patients demonstrated a response to MMF, although numbers were small (n = 4). We were not able to demonstrate a difference between responders and non-responders based on gender, age, previous therapies or time since diagnosis of ITP. Three of four previously splenectomized patients responded, two achieving complete response. We conclude that MMF is a useful steroid-sparing immunosuppressant to be considered in the second-line or later treatment of ITP.

Keywords: immune thrombocytopenia purpura; mycophenolate mofetil; platelets.

Publication types

  • Multicenter Study

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Combined Modality Therapy
  • Comorbidity
  • Drug Evaluation
  • Female
  • Humans
  • Immunoglobulins, Intravenous / therapeutic use
  • Immunosuppressive Agents / therapeutic use*
  • Male
  • Middle Aged
  • Mycophenolic Acid / analogs & derivatives*
  • Mycophenolic Acid / therapeutic use
  • Purpura, Thrombocytopenic, Idiopathic / drug therapy*
  • Purpura, Thrombocytopenic, Idiopathic / etiology
  • Purpura, Thrombocytopenic, Idiopathic / surgery
  • Remission Induction
  • Retrospective Studies
  • Salvage Therapy
  • Splenectomy
  • Virus Diseases / complications
  • Young Adult


  • Immunoglobulins, Intravenous
  • Immunosuppressive Agents
  • Mycophenolic Acid