The introduction of the JAK1 and JAK2 inhibitor ruxolitinib has significantly improved outcomes for some patients with myeloproliferative neoplasms (MPNs); however, the therapeutic armamentarium is still largely inadequate, researchers at the University of Florence, Italy, report in a study published in Blood.

The researchers note treatments have significantly improved for patients with myelofibrosis, extensive splenomegaly, and symptomatic burden with ruxolitinib. However, significant heterogeneity is still seen in patients with essential thrombocythemia (ET), polycythemia vera (PV), and primary myelofibrosis (PMF).

In their report, the researchers cite an urgent need for treatments to reduce cardiovascular complications, prevent hematological progression, and improve quality of life in patients with MPN.

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The researchers suggest that biological profiling techniques may soon be able to help better identify subgroups of patients with more homogeneous clinical behavior who might respond better to more tailored therapies.  They note that biological profiling could assist monitoring patients on therapy and could be incorporated into clinical trials.

A greater understanding of the molecular abnormalities and cellular pathways involved in the pathogenesis of MPN is leading to new approaches. Although new clinical trials with novel target drugs alone or in combination with ruxolitinib are underway, the researchers report that results from some early phase 1 and 2 clinical trials have been disappointing.


1. Vannucchi AM, Harrison CN. Emerging treatments for classical myeloproliferative neoplasms.  Blood.  2017;129(6):693-703.

This article originally appeared on ONA