Background:Polycythemia vera (PV) is associated with troublesome symptoms and reduced quality of life (QoL). Although its treatment is risk-adapted and aims to minimize or improve symptoms, symptom burden is not included as a risk stratification factor for PV. Symptom burden and its impact on QoL may be underestimated in "low risk" patients (with age <60 and without prior thrombo-hemorrhagic events).
Background. The national observational program MPN-QoL-2020 was focused on quality of life (QoL) and symptoms in patients with classical Ph-negative myeloproliferative neoplasms (MPNs) in the Russian Federation, as well as on the perception of the disease and treatment from the patient's and physician's perspective. Aim. To evaluate QoL in patients with different MPNs using new standardized questionnaires, to assess the most common symptoms and their impact on QoL in patients with myelofibrosis (MF), polycythemia vera (PV) and essential throm-bocythemia (ET), and to characterize the perception of the disease and treatment concerns from patients' perspective and their treating physicians' perspective. Materials & Methods. In total 1100 patients with MPNs (MF: n = 355, PV: n = 408, and ET: n = 337; mean age 58 ± 14 years; 61 % women) and 100 hematologists (mean age 42 ± 12 years; 85 % women) from 37 medical centers in 8 Federal districts of the Russian Federation participated in the study. All the patients filled out symptom assessment tool (MPN10), QoL questionnaire for patients with hematological nancies (HM-PRO) and patient's survey checklist; physicians filled out physician's survey checklist and patient record for each patient included in the study. Results. For the first time in Russia in a representative population of MPN patients in the real-world setting, QoL and symptom profiles in patients with different MPNs were characterized and symptom impact on the daily living of MPN patients was identified. MPN patients exhibited QoL impairment: noticeable detriments in physical and emotional functioning, as well as in eating and drinking regimen were found, social functioning was less impaired. More than one third of MPN patients had significant QoL impairment. The vast majority of patients experienced fatigue: 92.6 % MF patients, 83.7 % PI patients, and 82 % ET patients. Symptom prevalence severity differed across different MPNs. Top disease-related symptoms to be resolved were identified from patient's and physician's perspective. Discrepancies in the attitudes of MPN patients and their treating physicians to various aspects regarding the disease and its treatment were found as well as issues needed to be improved in the patient-physician communication were identified. Conclusion. The results of national research program MPN-QoL-2020 allowed to identify the areas of QoL impairment and symptom burden in MPN patients in Russia, to verify areas of concern related to the disease and its treatment in patients with different MPNs, as well as to highlight the unmet needs in this patients' population in our country. The outcomes of the study may contribute to establishing recommendations for improving/maintaining QoL in patients with MPNs and to developing measures aimed to raise awareness of this patients' population about the disease and its treatment.
Dasatinib as a front-line is a promising treatment option for CP-CML pts. Assessment of benefits and risks of this treatment regimen both from physician's and patient's perspective sounds worthy. We aimed to study QoL and clinical outcomes of dasatinib as a front-line treatment in CP CML pts in a real world setting. The total of 30 pts with CP CML (16 pts - TKI-naive, 14 pts - early switched to dasatinib after failure of imatinib treatment, median duration of imatinib treatment - 6 mths; switched pts) were involved in the multicenter observational prospective study. Median age - 47.6 y.o. (20-74), female - 33.3%; median disease duration - 5 mths; distribution of pts according to the Sokal score - 27.6% pts at low risk, 41.4% - at intermediate risk, 31% - at high risk. 27% pts had comorbidities: median Charlson Comorbidity Index was 3.0. All the pts filled out the QoL questionnaire SF-36 before dasatinib treatment, 3 and 12 mths after treatment start. Hematological, cytogenetic and molecular response rates (HR, CyR, MR) were registered after 3 and 12 mths of dasatinib treatment. Healthy controls (n=30) from QoL population normative database adjusted by age and gender to pts group were used for QoL comparison at baseline. Group comparisons were made using Mann-Whitney test, ANOVA and Wilcoxon matched pair test. QoL in CP CML pts at baseline was lower than in healthy controls: Integral QoL Index - 0.358 in pts vs 0.497 in healthy controls (p<0.05). Physical functioning in CP CML pts was significantly lower than in healthy controls: 78.8 vs 90.8 (p<0.05). No QoL differences were found between TKI-naive and switched pts before dasatinib treatment (p>0.05). During 12 months of dasatinib treatment positive changes in QoL were observed: Integral Quality of Life Index increased from 0.358 at baseline to 0.493 after 12 mths (p=0.05) and became comparable to healthy controls (p>0.05). Significant QoL improvement was registered in 12 mths of dasatinib treatment for role emotional (48.1 vs 81.5), role physical (44.4 vs 56.5) and social functioning(62.5 vs 80.6); p<0.05. Clinical outcomes were similar to the data of randomized clinical trials. After 3 mths of dasatinib treatment the majority of pts (92%) achieved complete HR, others had partial HR. After 12 mths of treatment 81.6% pts maintained or achieved complete HR; 69.2% pts exhibited complete CyR; 53.8% pts - major/complete MR. Four pts were dropped out of the study due to resistance to dasatinib (n=2) or due to severe AE (n=2). No cases of death were observed during the study. AE were similar to the ones registered within the clinical trials: in the most cases they were non-severe/moderate; severe AE (lung edema, bleeding from stomach ulcer) were observed in 2 pts; no cases of pleural effusion were revealed. In conclusion, patient-reported and clinical outcomes of dasatinib treatment in TKI-naïve pts and in pts early switched to dasatinib after failure of imatinib were obtained in a real world study. Significant QoL improvement in terms of role physical, role emotional and social functioning was observed. Clinical outcomes support the data obtained in clinical trials. Benefits and risks of front-line treatment with dasatinib in CML-CP both from physician's and patient's perspective were demonstrated in a real world setting. Disclosures Ionova: BMS: Research Funding; MSD: Speakers Bureau. Bulieva:BMS: Research Funding. Vinogradova:BMS: Research Funding. Kurbatova:BMS: Research Funding. Nikitina:BMS: Research Funding. Novitskaya:BMS: Research Funding. Rodionova:BMS: Research Funding. Usacheva:BMS: Research Funding. Chukavina:BMS: Research Funding. Shumkova:BMS: Research Funding.
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.
hi@scite.ai
10624 S. Eastern Ave., Ste. A-614
Henderson, NV 89052, USA
Copyright © 2024 scite LLC. All rights reserved.
Made with 💙 for researchers
Part of the Research Solutions Family.