Mycophenolate mofetil as a treatment for refractory idiopathic thrombocytopenic purpura

Abstract

Aim: To determine whether mycophenolate mofetil (MMF) has beneficial effects on refractory idiopathic thrombocytopenic purpura (ITP) and the corresponding cellular mechanism. Methods: Twenty refractory ITP patients resistant to corticosteroid and/or splenectomy and chemical therapy were given MMF 1.5‐2.0 g/d orally for a 2 to 4‐month period. Serum immunoglobulin was detected by rate nephelometry. Platelet‐associated antibodies (PAIgG) were assayed by enzymelinked immunosorbant assay. The immunophenotypic analysis was performed on a flow cytometer and cell apoptosis was detected with transferase mediated dUTP biotin nick end labeling (TUNEL) method. Results: Sixteen of the 20 (80%) patients had responses to MMF treatment; 9 (45%) achieved a complete response, 4 (20%) achieved a partial response, and 3 (15%) achieved a minor response. The therapeutic effects were found to be better in male patients than female patients. The number of CD3+ peripheral blood cells (PBCs) and CD4+ PBCs increased and the number of CD8+ PBCs decreased. The plasma level of IgG, IgM, IgA and platelet associated IgG (PAIgG) decreased in 86% of the patients. TUNEL assay showed that mycophenolate acid (MPA) 0.1 mmol/L induced apoptosis of peripheral blood mononuclear cells isolated from refractory ITP patients. The apoptosis rate was increased in male patients after treatment with MPA, but was unchanged in female patients. Conclusion: Therapy for a period of 8 to 16 weeks with mediandose of MMF was valuable for the treatment of refractory ITP.

Management of Chronic Childhood Immune Thrombocytopenic Purpura: AIEOP Consensus Guidelines

“…Treatment with mycophenolate has been reported in only 3 cases with chronic childhood ITP, without evident data about quality and duration of response [197,198,199,200,201,202,203,204]. …”

Autoimmunthrombozytopenie (AITP) des Erwachsenen: Klinik, Diagnose und Therapie

“…Eine große Anzahl anderer immunsuppressiver Substanzen wurde bei refraktärer AITP getestet wie Dapson [67,68], Cyclosporin [69], Mycophenolat [70][71][72], und andere. Der Stellenwert dieser Therapien bei splenektomierten Patienten mit schwerer refraktärer Thrombozytopenie ist jedoch ungewiss.…”

Multiagent induction and maintenance therapy for patients with refractory immune thrombocytopenic purpura (ITP)

“…[14][15][16] Many of the patients receiving maintenance treatment had received the combination treatment described in the first part of the study to acutely increase their platelet counts but also required ongoing treatment. The hypothesis tested in this section was that by combining 2 active agents with different mechanisms of effect and largely nonoverlapping toxicities, a higher response rate would be seen without an increase in toxicity.…”