An agenda for future research projects in polycythemia vera and essential thrombocythemia

he clinical course of essential thrombocythemia (ET) and polycythemia vera (PV) is characterized by an increased incidence of vascular complications and a tendency to progress to myelofibrosis (MF) or acute myeloid leukemia (AML). Over the past decade, new molecular and clinical knowledge in ET and PV has led to a significant improvement in the diagnostic, prognostic and therapeutic processes. Despite these advancements, many uncertainties remain concerning aspects of clinical decision-making. We identified some unmet needs in clinical practice and research that urgently require new scientific initiatives. For each of these, we reviewed the most significant existing evidence and made proposals for translational and clinical investigations. We acknowledge that several other clinically relevant unmet needs in the management of patients with PV and ET remain. These could not be addressed due to space constrains, and include, above all, prediction of evolution to secondary forms of myelofibrosis, identification of genetic predictors of survival and of specific subgroups of patients to include in intervention trials with novel drugs that are claimed to modify disease course.