Essential thrombocythemia: challenges in clinical practice and future prospects

Abstract

Essential thrombocythemia (ET) was described in 1934 and in the intervening time progress has been achieved in better understanding molecular pathogenesis and which patients may have greatest risk of progression or vascular events. However it has been over a decade since a new therapy has been approved for this disease. We are beginning to understand more fully both the heterogeneity of this disease, which is largely driven by driver mutation status, as well as the impact upon patients of disease-related symptoms such as fatigue. In this review we provide a practical overview of diagnosis and management of ET with a focus upon difficult patient scenarios and some consideration of what comprehensive care might require. Finally, we also discuss newer therapies and how these might be assessed.