JAK Inhibition for the Treatment of Myelofibrosis: Limitations and Future Perspectives.

ABSTRACT: The 2011 approval of ruxolitinib ushered in the Janus kinase (JAK) inhibitor era in the treatment of myelofibrosis (MF), and 2019 saw the US approval of fedratinib. The first therapeutic agents approved by regulatory authorities for MF, these drugs attenuate the overactive JAK-signal transducer and activator of transcription (STAT) signaling universally present in these patients, translating into major clinical benefits in terms of spleen shrinkage and symptom improvement. These, in turn, confer a survival advantage on patients with advanced disease, demonstrated in the case of ruxolitinib, for which long-term follow-up data are available. However, JAK inhibitors do not improve cytopenias in most patients, have relatively modest effects on bone marrow fibrosis and driver mutation allele bu

SUBMITTER: Bose P 

PROVIDER: S-EPMC7375176 | biostudies-literature | 2020 Aug

REPOSITORIES: biostudies-literature