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- W2291022020 abstract "BCR-ABL negative myeloproliferative neoplasms (MPN’s) include essential thrombocythemia (ET), polycythemia vera (PV) and primary myelofibrosis (PMF), the latter notable for harboring the greatest morbidity and mortality amongst the subclass. The complex biogenetic underscoring of these clonal hemopathies has historically stifled meaningful advances in gene-targeted therapies and compromised inhibition of the pro-inflammatory cytokines foundational to symptom development. The paramount discovery of the JAK2V617F mutation led to the integration of genetic biomarkers into diagnostic and treatment strategies. This past decade has since been marked by the rapid development of JAK2 inhibitor therapies capable of reducing splenomegaly, cytopenias and constitutional symptoms with minimal myelosuppression and secondary toxicities. Ruxolitinib was the first among JAK inhibitors to achieve FDA approval in 2011 for intermediate- and high-risk myelofibrosis. Notable advances in our understanding of this therapies efficacy, tolerability and impact on life-expectancy have been made within the past year, including post-hoc analysis’ of the COMFORT-I and COMFORTII trials, drug impact despite baseline thrombocytopenias and effects on JAK2V617F mutational status. Integrating scientific advancement into clinical practice remains a challenge for all MF providers. The recent development of Individual Patient Supply Programs (IPSP’s) and Compassionate Use Programs (CUP’s) has extended meaningful and efficacious therapies to patients that otherwise may not have access outside the clinical trial setting. As we survey the future of MPN treatments, it is clear that integration of scientific advancements must parallel timesensitive patient application so as to offer the greatest relief to those afflicted by the MPN disease burden." @default.
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- W2291022020 date "2013-01-01" @default.
- W2291022020 modified "2023-09-23" @default.
- W2291022020 title "JAK-2 Inhibition: Current Roles in Myelofibrosis and Initial Lessons Learned from Mexico" @default.
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