LAUSR

ABSTRACT Introduction: The outlook for patients with myeloproliferative neoplasms, particularly myelofibrosis, has improved in recent years, with greater understanding of the pathogenesis and the subsequent development of a plethora of new agents. Areas covered: This article will discuss some of the advances in the field in recent years and explore in greater detail some of the most advanced emerging agents as well as those with greatest potential. An extensive literature review has been performed to identify recent clinical trials and any relevant pre-clinical work. Expert commentary: Important discoveries regarding molecular pathogenesis have led to advances in diagnostic algorithms, prognosis and ultimately also treatment strategies. However, the therapeutic armamentarium for MPN is still largely inadequate to cope with significant challenges including normalization of life span, reduction of cardiovascular complications, prevention of hematological progression and improved quality of life. Sadly, no currently available drugs have shown clear evidence of disease-modifying activity and results of early phase I and II clinical trials have been quite disappointing to date, with toxicities sometimes limiting and a lack of meaningful biological surrogate end points.