Therapeutic efficacy and clinical effectiveness of mycophenolate mofetil and dexamethasone for immune thrombocytopenia: A retrospective observational study

BACKGROUND: Immune thrombocytopenia (ITP) is an autoimmune hemorrhagic disorder, where autoreactive T-cells and/or autoantibodies destroy platelets and megakaryocytes in the spleen and bone marrow, respectively. To the best of our knowledge, this is the largest cohort of cases wherein patients were treated with novel combination therapy of a corticosteroid with an adjunct immunosuppressive agent for the treatment of ITP in adults. In cohort of 23 patients, 11 patients have no response and 12 patients have shown partial to complete response (CR) to the treatment. The primary aim of the present study was to explore the safety and efficacy of combination therapy in chronic ITP along by evaluating the toxicity associated with prolonged steroid exposure. The secondary aim was to compare the cost benefit with other available modalities for chronic ITP treatment. MATERIALS AND METHODS: A retrospective observational study was carried out by collecting data from electronic medical records of all the ITP patients treated at HCG Manavata Cancer Centre, India, between May 1, 2019, and April 30, 2020. Inclusion and exclusion criteria were strictly followed for data collection and analysis. RESULTS: Twelve (52%) of the 23 patients have shown response to the combinational therapy; 5 (22%) patients achieved a partial response (PR) and 7 (30%) achieved a CR. In the PR group, 3 patients developed thrombocytopenia and 1 switched to thrombopoietin receptor agonists, whereas in 7 CR patients, 6 have maintained it until end and 1 patient was switched to maintenance therapy. CONCLUSION: A combination of immunosuppressant and corticosteroid on ITP patients appeared to be effective, tolerable, with minimal adverse side effects, and an economical alternative. Therefore, this novel combination therapy may be an excellent alternative for the treatment of patients with ITP in clinical settings.