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      The Myeloproliferative Neoplasm Landscape: A Patient’s Eye View

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          Abstract

          Patients with myeloproliferative neoplasms (MPNs), a group of rare haematological conditions including polycythaemia vera, essential thrombocythaemia, and myelofibrosis, often experience a range of symptoms which can significantly impact their quality of life (QoL). Although symptom burden is highest in myelofibrosis and high-risk patients, lower-risk patients also report symptoms impacting their daily life and ability to work. In addition to physical symptoms, MPNs affect emotional well-being, with anxiety and depression frequently reported by patients. Despite significant advances in treatment options, such as the introduction of JAK1/JAK2 inhibitors, therapy for MPNs is often palliative; therefore, reduction of symptoms and improvement of QoL should be considered as major treatment goals. One of the main issues impacting MPN treatment is the discord between patient and physician perceptions of symptom burden, treatment goals, and expectations. New technologies, such as app-based reporting, can aid this communication, but are still not widely implemented. Additionally, regional variation further affects the psychosocial burden of MPNs on patients and their associates, as treatments and access to clinical trials are options for patients living in some areas, but not others. Overcoming some of the challenges in patient–physician communication and treatment access are key to improving disease management and QoL, as well as giving the patient greater input in treatment decisions.

          Plain Language Summary

          Myeloproliferative neoplasms (MPNs) are a group of blood diseases where the body makes too many blood cells. Patients with MPNs can have symptoms which interfere with their daily lives, such as tiredness, pain, sweating at night, dizziness, itching, and difficulty sleeping. They also often suffer from anxiety and/or depression. In nearly all cases, physicians cannot cure the disease, but drugs can prevent blood clots and reduce the speed at which the disease gets worse. Usually, the main aim of treatment is improving patients’ quality of life (QoL). Targeted drugs, such as ruxolitinib, treat MPNs and reduce symptoms, but do not cure the disease. Patients frequently want to play a bigger part in decisions about their treatment. However, physicians and patients often have different views on how well treatments are working and what to expect from the treatment. This can mean that patients feel they are not getting the best treatment for their symptoms. Also, patients may not be able to get some treatments or take part in a trial of a new drug, depending on where they live. This creates feelings of unfairness which can affect their mental health. Addressing all these problems may help improve the QoL for patients with these blood diseases.

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          Most cited references42

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          Long-term survival and blast transformation in molecularly annotated essential thrombocythemia, polycythemia vera, and myelofibrosis.

          Janus kinase 2 (JAK2) mutations define polycythemia vera (PV). Calreticulin (CALR) and myeloproliferative leukemia virus oncogene (MPL) mutations are specific to JAK2-unmutated essential thrombocythemia (ET) and primary myelofibrosis (PMF). We examined the effect of these mutations on long-term disease outcome. One thousand five hundred eighty-one patients from the Mayo Clinic (n = 826) and Italy (n = 755) were studied. Fifty-eight percent of Mayo patients were followed until death; median survivals were 19.8 years in ET (n = 292), 13.5 PV (n = 267; hazard ratio [HR], 1.8; 95% confidence interval [CI], 1.4-2.2), and 5.9 PMF (n = 267; HR, 4.5; 95% CI, 3.5-5.7). The survival advantage of ET over PV was not affected by JAK2/CALR/MPL mutational status. Survival in ET was inferior to the age- and sex-matched US population (P < .001). In PMF (n = 428), but not in ET (n = 576), survival and blast transformation (BT) were significantly affected by mutational status; outcome was best in CALR-mutated and worst in triple-negative patients: median survival, 16 vs 2.3 years (HR, 5.1; 95% CI, 3.2-8.0) and BT, 6.5% vs 25% (HR, 7.6; 95% CI, 2.8-20.2), respectively. We conclude that life expectancy in morphologically defined ET is significantly reduced but remains superior to that of PV, regardless of mutational status. In PMF, JAK2/CALR/MPL mutational status is prognostically informative.
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            Safety and Efficacy of Fedratinib in Patients With Primary or Secondary Myelofibrosis: A Randomized Clinical Trial.

            Myelofibrosis (MF) is a BCR-ABL-negative myeloproliferative neoplasm characterized by anemia, splenomegaly, debilitating constitutional symptoms, and shortened survival. Fedratinib, a JAK2-selective inhibitor, previously demonstrated clinically beneficial activity in patients with MF in early-phase trials.
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              Myeloproliferative Neoplasms.

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                Author and article information

                Contributors
                cmpnrf@gmail.com
                Journal
                Adv Ther
                Adv Ther
                Advances in Therapy
                Springer Healthcare (Cheshire )
                0741-238X
                1865-8652
                23 April 2020
                23 April 2020
                2020
                : 37
                : 5
                : 2050-2070
                Affiliations
                [1 ]Canadian MPN Research Foundation, Edmonton, AB Canada
                [2 ]MPN Voice, London, UK
                Article
                1314
                10.1007/s12325-020-01314-0
                7467498
                32329011
                7d80e796-21f9-4e69-b8d9-68a2aad544ca
                © The Author(s) 2020

                Open AccessThis article is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License, which permits any non-commercial use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by-nc/4.0/.

                History
                : 10 February 2020
                Funding
                Funded by: FundRef http://dx.doi.org/10.13039/100008272, Novartis Pharmaceuticals 12325Corporation;
                Categories
                Review
                Custom metadata
                © Springer Healthcare Ltd., part of Springer Nature 2020

                jak1/jak2 inhibitors,myeloproliferative neoplasms,patient-reported outcomes,quality of life,regionality,unmet needs

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