Myeloproliferative neoplasms (MPNs) have a significant impact on patients’ overall health and productivity: the MPN Landmark survey

Abstract

BackgroundThe Philadelphia chromosome−negative myeloproliferative neoplasms (MPN) myelofibrosis (MF), polycythemia vera (PV), and essential thrombocythemia (ET) negatively affect patient quality of life (QoL) and are associated with increased risk of mortality.MethodsThe MPN Landmark survey was conducted from May to July 2014 in patients with MF, PV, or ET under active management in the United States. The survey assessed respondent perceptions of disease burden and treatment management and included questions on overall disease burden, QoL, activities of daily living, and work productivity. Outcomes were further analyzed by calculated (ie, not respondent-reported) prognostic risk score and symptom severity quartile.ResultsThe survey was completed by 813 respondents (MF, n = 207; PV, n = 380; ET, n = 226). The median respondent age in each of the 3 MPN subtypes ranged from 62 to 66 years; median disease duration was 4 to 7 years. Many respondents reported that they had experienced MPN-related symptoms ≥1 year before diagnosis (MF, 49 %; PV, 61 %; ET, 58 %). Respondents also reported that MPN-related symptoms reduced their QoL, including respondents with low prognostic risk scores (MF, 67 %; PV, 62 %; ET, 57 %) and low symptom severity (MF, 51 %; PV, 33 %; ET, 15 %). Many respondents, including those with a low prognostic risk score, reported that their MPN had caused them to cancel planned activities or call in sick to work at least once in the preceding 30 days (cancel planned activities: MF, 56 %; PV, 35 %; ET, 35 %; call in sick: MF, 40 %; PV, 21 %; ET, 23 %).ConclusionsThese findings of the MPN Landmark survey support previous research about the symptom burden experienced by patients with MPNs and are the first to detail the challenges that patients with MPNs experience related to reductions in activities of daily living and work productivity.Electronic supplementary materialThe online version of this article (doi:10.1186/s12885-016-2208-2) contains supplementary material, which is available to authorized users.

Ruxolitinib vs best available therapy for ET intolerant or resistant to hydroxycarbamide

“…32 The symptom response rate, defined as a 50% reduction in TSS, during the first 12 months did not significantly differ between the 2 arms. However, maximum percentage TSS reduction during the first 12 months of treatment was significantly greater for ruxolitinib compared with BAT and was more rapid in the ruxolitinib arm.…”

“…Overall, the majority, 79% (41/52), of BAT patients received 1 or both agents before or during the study. On-study BAT included in addition busulfan 32 P and HC; several international guidelines recommend busulfan or 32 P for older patients.…”

From leeches to personalized medicine: evolving concepts in the management of polycythemia vera

“…In a recent landmark study into MPN in the United States that interviewed 380 PV patients undergoing treatment, fatigue and itching were identified by 33% and 9% of the respondents as the symptom they most urgently wanted to resolve. 64 The pathogenetic link between symptoms and clonal myeloproliferation is likely sustained by an abnormal release and signaling of inflammatory cytokines through the deranged JAK/STAT pathway, 65 a concept that is reinforced by the unique and rapid symptomatic efficacy of the JAK1 and JAK2 inhibitor ruxolitinib. 66 To add to the burden associated with PV, one must consider the side effects of treatment, including worsening of fatigue and other signs of iron deficiency in heavily phlebotomized patients, varying manifestations of intolerance to hydroxyurea, the known toxicities of interferon, the increased Herpes Zoster reactivation with ruxolitinib, to name but a few.…”

Diagnostik und Therapie der Polycythaemia vera im Jahre 2015

“…1). Die Resultate machen deutlich, dass der durchschnittliche Schweregrad zwar relativ moderat ist, dass aber nicht wenige Patienten an mehreren Symptomen leiden, was die Lebensqualität erheblich beeinflussen kann [29,30]. Insbesondere der oft quälende und nicht selten therapierefraktäre Pruritus stellt für manche Patienten ein die Lebensqualität und den Aktionsradius massiv beeinträchtigendes Symptom dar.…”