Mycophenolate mofetil therapy for severe immune thrombocytopenia

Abstract

*We regret to note that Dr Roberto Stasi, one of our co-authors, has very sadly passed away since the time of original data collection. We would like to acknowledge his valuable contribution to this work SummarySevere 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 52Á5 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.

Mycophenolate mofetil for the treatment of children with immune thrombocytopenia and Evans syndrome. A retrospective data review from the Italian association of paediatric haematology/oncology

“…This favourable outcome is in agreement with the results from a recently published series of 46 adults treated with MMF for ITP (Taylor et al, 2015). This favourable outcome is in agreement with the results from a recently published series of 46 adults treated with MMF for ITP (Taylor et al, 2015).…”

Characteristics, outcome, and response to therapy of multirefractory chronic immune thrombocytopenia

“…Our findings contrast with several reports or clinical studies showing prolonged response with such therapies, but patients showed less refractory disease than our patients. [13][14][15][16][17][18][19] Despite the efficacy of HSCT, the high risk of mortality associated with this procedure (1 death in our cohort) argues for its proposal for patients with no alternative therapeutic option only. However, the combination of Tpo-RAs with immunosuppressant drugs was effective with an overall response rate (R 1 CR) of 70% in the 10 patients who received this treatment.…”

How I treat refractory immune thrombocytopenia

“…[76][77][78][79][80][81][82] We begin MMF at a dose of 500 mg orally twice per day and increase the dose to 1000 to 1500 mg twice per day after 2 weeks. Protocols that use this approach have demonstrated overall response rates of 50% to 60%.…”

“…Protocols that use this approach have demonstrated overall response rates of 50% to 60%. [76][77][78][79][80][81] Durability of response after discontinuation of therapy is variable. 77,79,82 MMF is generally well tolerated; principal adverse effects include headache and gastrointestinal symptoms.…”