title: Mycophenolate mofetil therapy for severe immune thrombocytopenia.
abstract: 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.
authors.affiliation: University College London Hospitals NHS Foundation Trust, London, UK.
authors.forename: Alice
authors.initials: A
authors.lastname: Taylor
authors.affiliation: Imperial College Healthcare NHS Trust, London, UK.
authors.forename: Lucy
authors.initials: L
authors.lastname: Neave
authors.affiliation: St George's University Hospitals NHS Foundation Trust, London, UK.
authors.forename: Shalini
authors.initials: S
authors.lastname: Solanki
authors.affiliation: University College London Hospitals NHS Foundation Trust, London, UK.
authors.forename: John Paul
authors.initials: JP
authors.lastname: Westwood
authors.affiliation: Imperial College Healthcare NHS Trust, London, UK.
authors.forename: Ilaria
authors.initials: I
authors.lastname: Terrinonive
authors.affiliation: University College London Hospitals NHS Foundation Trust, London, UK.
authors.forename: Siobhan
authors.initials: S
authors.lastname: McGuckin
authors.affiliation: University College London Hospitals NHS Foundation Trust, London, UK.
authors.forename: Jaimal
authors.initials: J
authors.lastname: Kothari
authors.affiliation: Imperial College Healthcare NHS Trust, London, UK.
authors.forename: Nichola
authors.initials: N
authors.lastname: Cooper
authors.affiliation: St George's University Hospitals NHS Foundation Trust, London, UK.
authors.forename: Roberto
authors.initials: R
authors.lastname: Stasi
authors.affiliation: University College London Hospitals NHS Foundation Trust, London, UK.
authors.forename: Marie
authors.initials: M
authors.lastname: Scully
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D003131:Combined Modality Therapy; D015897:Comorbidity; D004341:Drug Evaluation; D005260:Female; D006801:Humans; D016756:Immunoglobulins, Intravenous / Q000627:therapeutic use; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D009173:Mycophenolic Acid / Q000031:analogs & derivatives* / Q000627:therapeutic use; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy* / Q000209:etiology / Q000601:surgery; D012074:Remission Induction; D012189:Retrospective Studies; D016879:Salvage Therapy; D013156:Splenectomy; D014777:Virus Diseases / Q000150:complications; D055815:Young Adult
chemical_list: D016756:Immunoglobulins, Intravenous; D007166:Immunosuppressive Agents; D009173:Mycophenolic Acid
keywords: immune thrombocytopenia purpura; mycophenolate mofetil; platelets