Integrating novel therapeutic strategies in myeloproliferative neoplasms: Driving disease-directed progress

Abstract

Over the past decade, there has been a substantial increase in the diversity and number of therapeutic options for myeloproliferative neoplasms (MPNs). While many remain within the clinical trial arena, the clinician and patient community have seen more approvals reaching the clinic and a rethink on how best we should be approaching these disorders is required. In parallel, there needs to be a paradigm shift in end-point considerations within MPN clinical trial design, with a growing emphasis on longer term disease stability and modification, molecular responses, histomorphological modification, reductions in complications (e.g. thrombotic complications or disease transformation) and improved survival alongside the conventional goals such as haematological responses and symptom and spleen improvements where relevant. Given the heterogeneity of these disorders, with often marked intrapatient variability, greater consideration must be given to optimised sequencing strategies and rational combination approaches and, importantly, an emphasis on patient-specific trajectories. In this review, we appraise the contemporary therapeutic armamentarium across the MPN spectrum and explore how these advances may best facilitate increasing personalised approaches in an evolving and complex treatment landscape.