Controversies in the treatment of immune thrombocytopenia

Abstract

We address three current controversies in management of immune thrombocytopenia (ITP): Should asymptomatic children with newly diagnosed ITP and severe thrombocytopenia be treated? Does intensification of up-front therapy in adults with newly diagnosed ITP impact long-term outcomes? Is splenectomy still the second-line treatment of choice in adults with chronic ITP? Severe bleeding is rare in children with ITP. There is little evidence that the platelet count predicts or that treatment prevents severe bleeding in this population. Intensified treatment with high-dose dexamethasone and rituximab in adults with newly diagnosed ITP is associated with improved platelet responses at 6 and 12 months but greater toxicity compared with standard therapy. Rituximab and thrombopoietin receptor agonists have emerged as suitable alternatives to splenectomy for second-line management of adults with chronic ITP. We generally observe children with newly diagnosed ITP and mild or no bleeding symptoms, irrespective of platelet count. We do not routinely use intensified up-front therapy in adults with newly diagnosed ITP. We discuss the advantages and disadvantages of splenectomy, rituximab, and thrombopoietin receptor agonists with our patients and make a joint decision that takes into consideration age, comorbidities, lifestyle, values, preferences, and financial considerations.