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Abstract
Philadelphia chromosome-negative myeloproliferative neoplasms (MPNs) are clonal stem cell-derived malignancies that include primary myelofibrosis, polycythemia vera and essential thrombocythemia and are characterized by dysregulated Janus kinase–signal transducers and activators of transcription (JAK–STAT) signaling. Clinical manifestations include splenomegaly, cytopenias and/or systemic inflammation. Patients have a heterogeneous symptom profile that includes fatigue, loss of appetite, pruritus and night sweats, which significantly impact quality of life (QoL) and lead to poor survival outcomes. With the introduction of JAK inhibitors, improvement in disease-related symptoms has emerged as a realistic expectation of therapy and an integral measure of clinical efficacy. The JAK1/JAK2 inhibitor ruxolitinib is approved for the treatment of myelofibrosis and is currently under clinical development for polycythemia vera. Ruxolitinib has demonstrated significant reductions in symptom burden, with consequent improvements in QoL measures. With the potential to improve QoL, recognition of the impact and burden of symptoms on patients with MPNs is critical.
Acknowledgments
Medical writing support, funded by Incyte Corporation, was provided by Tara Beers Gibson, PhD, CMPP, and Susan Sutch, PharmD, CMPP, of Evidence Scientific Solutions.
Potential conflict of interest
Disclosure forms provided by the authors are available with the full text of this article at www.informahealthcare.com/lal.