Abstract:BACKGROUND:
The presence of constitutional symptoms has been associated with increased mortality risk in myelofibrosis (MF) (Blood 2010;115(9):1703-8). New therapies exist which alleviate the severe symptom burden profile observed in MF patients but are only approved for use in those with intermediate-2 or high risk disease (N Engl J Med 2012;366:787-798). However, it has been proposed that there are patients who may benefit from symptom based treatment regardless of prognostic score (Am Soc Hem… Show more
“…The severity score for the overall MPN population was > 5 for 24 of 31 individual symptoms assessed. Scores > 5 for individual symptoms in the MPN-SAF TSS have been associated with advanced disease and the need for therapy [ 28 , 29 ]. Fig.…”
Myelofibrosis (MF), polycythemia vera (PV), and essential thrombocythemia (ET) are myeloproliferative neoplasms (MPNs) associated with high disease burden, reduced quality of life (QOL), and shortened survival. To assess how MPNs affect patients, we conducted a global MPN Landmark survey. This online survey of patients with MPNs and physicians was conducted in Australia, Canada, Germany, Japan, Italy, and the United Kingdom. The survey measured MPN-related symptoms and the impact of MPNs on QOL and the ability to work as well as disease-management strategies. Overall, 219 physicians and 699 patients (MF, n = 174; PV, n = 223; ET, n = 302) completed the survey; 90% of patients experienced MPN-related symptoms. The most frequent and severe symptom was fatigue. Most patients experienced a reduction in QOL, including those with low symptom burden or low-risk scores. A substantial proportion of patients reported impairment at work and in overall activity. Interestingly, physician feedback and blood counts were the most important indicators of treatment success among patients, with improvements in symptoms and QOL being less important. Regarding disease management, our study revealed a lack of alignment between physician and patient perceptions relating to communication and disease management, with patients often having different treatment goals than physicians. Overall, our study suggested that therapies that reduce symptom burden and improve QOL in patients with MPNs are crucial in minimizing disease impact on patient daily lives. Additionally, our findings showed a need for improved patient-physician communication, standardized monitoring of symptoms, and agreement on treatment goals.
“…The severity score for the overall MPN population was > 5 for 24 of 31 individual symptoms assessed. Scores > 5 for individual symptoms in the MPN-SAF TSS have been associated with advanced disease and the need for therapy [ 28 , 29 ]. Fig.…”
Myelofibrosis (MF), polycythemia vera (PV), and essential thrombocythemia (ET) are myeloproliferative neoplasms (MPNs) associated with high disease burden, reduced quality of life (QOL), and shortened survival. To assess how MPNs affect patients, we conducted a global MPN Landmark survey. This online survey of patients with MPNs and physicians was conducted in Australia, Canada, Germany, Japan, Italy, and the United Kingdom. The survey measured MPN-related symptoms and the impact of MPNs on QOL and the ability to work as well as disease-management strategies. Overall, 219 physicians and 699 patients (MF, n = 174; PV, n = 223; ET, n = 302) completed the survey; 90% of patients experienced MPN-related symptoms. The most frequent and severe symptom was fatigue. Most patients experienced a reduction in QOL, including those with low symptom burden or low-risk scores. A substantial proportion of patients reported impairment at work and in overall activity. Interestingly, physician feedback and blood counts were the most important indicators of treatment success among patients, with improvements in symptoms and QOL being less important. Regarding disease management, our study revealed a lack of alignment between physician and patient perceptions relating to communication and disease management, with patients often having different treatment goals than physicians. Overall, our study suggested that therapies that reduce symptom burden and improve QOL in patients with MPNs are crucial in minimizing disease impact on patient daily lives. Additionally, our findings showed a need for improved patient-physician communication, standardized monitoring of symptoms, and agreement on treatment goals.
“…Patients with PV or ET show near-normal life expectancies, but the median survival of patients with PMF ranges from 4 to 5.5 years. The majority of patients experience at least one of the symptoms caused by cytopenia, splenomegaly, and increased proinflammatory cytokine levels [ 80 , 81 ]. The Myeloproliferative Neoplasm Symptom Assessment Form total symptom score is a simple assessment tool for checking a patient’s constitutional symptoms, splenomegaly related symptoms, and quality of life at diagnosis and during the course of treatment [ 82 , 83 ].…”
In 2016, the World Health Organization (WHO) revised the diagnostic criteria of myeloproliferative neoplasms (MPNs) based on the discovery of disease-driving genetic aberrations and extensive analysis of the clinical characteristics of MPN patients. Recent studies have suggested that additional somatic mutations have clinical impacts on the prognosis of patients harboring these genetic abnormalities. Treatment strategies have also advanced with the introduction of JAK inhibitors, one of which has been approved for the treatment of myelofibrosis patients and hydroxyurea-resistant or intolerant polycythemia vera patients. Because recently developed drugs aim not only at hematologic responses but also at symptomatic and molecular responses, the response criteria were refined as well. Based on these changes, we have revised the guidelines by presenting the diagnosis, treatment, and risk stratification of MPNs that fit the reality in Korea.
“…7 An MPN Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) ≥20, a worst individual item score > 5, or combined criteria of both warrant treatment. 8 There has been a lack of estimates for the incidence/prevalence and treatment patterns of MPNs in many regions of the world, including countries from South Asia, Asia Pacific, Middle East, and Turkey, leading to a difficulty in estimating the true disease burden. Hence, there is a need to establish databases like registries, which would provide information on the "real-world" data in these regions.…”
Philadelphia chromosome-negative (Ph−) myeloproliferative neoplasms (MPNs) are a heterogeneous group of clonal disorders of the bone marrow, and are associated with a high disease burden, reduced quality of life (QOL), and shortened survival. This multinational, multicenter, non-interventional registry "MERGE" was initiated with an objective to collect data on the epidemiological indices of classical Ph-MPNs, existing treatment patterns, and impact of MPNs on health-related QOL in various countries/regions in Asia, including the Middle East, Turkey, and Algeria.Of the 884 eligible patients with MPNs, 169 had myelofibrosis (MF), 301 had polycythemia vera (PV), 373 had essential thrombocythemia (ET), and 41 had unclassified MPNs. The median age was 58 years (range, 47-66 years), and 50% of patients were males. The prevalence and incidence of MPNs were estimated to be 57-81 and
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.
