Patient-Reported Outcomes Data From REVEAL at the Time of Enrollment (Baseline): A Prospective Observational Study of Patients With Polycythemia Vera in the United States

Ruben Mesa, Ralph V Boccia, Michael R Grunwald, Stephen T Oh, Philomena Colucci, Dilan Paranagama, Shreekant Parasuraman, Brady L Stein, Ruben Mesa, Ralph V Boccia, Michael R Grunwald, Stephen T Oh, Philomena Colucci, Dilan Paranagama, Shreekant Parasuraman, Brady L Stein

Abstract

Background: Patients with polycythemia vera (PV) often experience symptoms that adversely affect their quality of life (QoL). The ongoing, prospective, observational REVEAL (Prospective Observational Study of Patients With Polycythemia Vera in US Clinical Practices) study was designed to collect contemporary data regarding burden of disease, clinical management, patient-reported outcomes (PROs), and health care resource utilization from adult patients with PV in the United States.

Patients and methods: Data on PROs were collected at enrollment using the Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS; range, 0-100); the European Organization for Research and Treatment of Cancer-Core Quality of Life Questionnaire, version 3.0 (EORTC QLQ-C30; range, 0-100); and the Work Productivity and Activity Impairment Questionnaire-Specific Health Problem (WPAI-SHP; range, 0%-100%).

Results: Among 2309 patients, mean (SD) disease duration was 5.8 (6.1) years and Charlson Comorbidity Index was 3.4 (0.8); 54.0% (1247/2309) were male. Mean (SD) MPN-SAF TSS was 18.8 (15.5). The most common symptoms were fatigue (80.1% [1844/2302]), early satiety (60.9% [1402/2302]), and inactivity (57.6% [1324/2302]). The most common severe symptoms were fatigue (16.8% [387/2302]), itching (13.4% [308/2302]), and inactivity (11.8% [271/2302]). The mean (SD) EORTC QLQ-C30 global health status/QoL score was 73.1 (23.2): mean functional subscale scores ranged from 80.5 (23.9) for cognitive functioning to 85.7 (24.6) for social functioning. The mean WPAI-SHP activity impairment score was 19.7% (n = 2300). Employed patients had mean WPAI-SHP scores for absenteeism, presenteeism, and overall work impairment of 3.2% (n = 810), 12.1% (n = 807), and 13.4% (n = 802), respectively.

Conclusion: These data confirm that many patients with PV experience symptoms, QoL impairments, and work productivity impairments that negatively affect their lives. Longitudinal data from REVEAL will be important for evaluating how PROs change over time in these patients.

Trial registration: ClinicalTrials.gov NCT02252159.

Keywords: Activity impairment; Myeloproliferative neoplasm; Quality of life; Symptoms; Work productivity.

Copyright © 2018 The Authors. Published by Elsevier Inc. All rights reserved.

Figures

Figure 1.. Myeloproliferative Neoplasm Symptom Assessment From…
Figure 1.. Myeloproliferative Neoplasm Symptom Assessment From (MPN-SAF) Sympton Severity at Enrollment. aWeight Loss Indicate Unintentional Weight Loss in the Past 6 Months
aEach item was scored on a scale from 0 (absent) to 10 (worst imaginable). Only evaluable patients with MPN-SAF data (n = 2302) were included.
Figure 2. Mean (SD) Myeloproliferative Neoplasm Symptom…
Figure 2. Mean (SD) Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) Symptom Scores at Enrollmenta
a Each item was scored on a scale from 0 (absent) to 10 (worst imaginable). Only evaluable patients with MPN-SAF data (n = 2302) were included. b Unintentional weight loss in the past 6 months.
Figure 3. Mean European Organization for Research…
Figure 3. Mean European Organization for Research and Treatment of Cancer Core Quality-of-Life Questionnaire (EORTC QLQ-C30) Scores for (A) Symptom Subscales and (B) QoL and Functional Subscales at Enrollmenta
Abbreviation: QoL, quality of life. a All scores were standardized using linear transformation to 0 to 100. Only evaluable patients with EORTC QLQ-C30 data (n = 2298–2304 for each score) were included.
Figure 4. Mean Work Productivity and Activity…
Figure 4. Mean Work Productivity and Activity Impairment Questionnaire—Specific Health Problem Scores at Enrollmenta
Abbreviation: PV polycythemia vera. a All items on the basis of 7-day recall (ie, regarding work/activity during the 7 days preceding the survey).

References

    1. Arber DA, Orazi A, Hasserjian R, et al. The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood 2016; 127:2391405.
    1. Hultcrantz M, Kristinsson SY, Andersson TM, et al. Patterns Of survival among patients with myeloproliferative neoplasms diagnosed in Sweden from 1973 to a population-based study. J Clin Oncol 2012; 30:2995–3001.
    1. Emanuel RM, Dueck AC, Geyer HL, et al. Myeloproliferative Neoplasm (MPN) Symptom Assessment Form Total Symptom Score: prospective international assessment Of an abbreviated symptom burden scoring system among patients with MPNs. J Clin Oncol 2012; 30:4098–103.
    1. Johansson P, Mesa R, Scherber R, et al. Association between quality Of life and clinical parameters in patients with myeloproliferative neoplasms. Leuk Lymphoma 2012; 53:441–4.
    1. Mesa R, Miller CB, Thyne M, et al. Myeloproliferative neoplasms (MPNs) have a significant impact on patients’ overall health and productivity: the MPN Landmark survey. BMG Cancer 2016; 16:167.
    1. Scherber R, Dueck AC, Johansson P, et al. The Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF): international prospective validation and reliability trial in 402 patients. Blood 2011; 118:401–8.
    1. Mesa IRA, Niblack J, Wadleigh M, et al. The burden of fatigue and quality Of life in myeloproliferative disorders (MPDs): an international internet-based survey Of 1179 MPD patients. Cancer 2007; 109:68–76.
    1. Geyer H, Scherber R, Kosiorek H, et al. Symptomatic profiles Of patients with polycythemia Vera: implications of inadequately controlled disease. J Clin Oncol 2016; 34:151–9.
    1. Gruppo Italiano Studio Policitemia. Polycythemia Vera: the natural history of 1213 patients followed for 20 years. Gruppo Italiano Studio Policitemia. Ann Intern Med 1995; 123:656–64.
    1. Tefferi A, Rumi E, Finazzi G, et al. Survival and prognosis among 1545 patients with contemporary polycythemia Vera: an international study. Leukemia 2013; 27: 1874–81.
    1. Abelsson J, Andrexsson B, Samuelsson J, et al. Patients with polycythemia vera have the worst impairment of quality of life among patients with newly diagnosed myeloproliferative neoplasms. Leuk Lymphoma 2013; 54:2226–30.
    1. Stein BL, Williams DM, Wang NY, et al. sex differences in the JAK2 V617F allele burden in chronic myeloproliferative disorders. Haematologica 2010; 95:1090–7.
    1. Stein BL, Saraf S, Sobol U, et al. Age-related differences in disease characteristics and clinical outcomes in polycythemia vera. Leuk Lymphoma 2013; 54:1989–95.
    1. National Comprehensive Cancer Network. clinical practice guidelines in oncology. Myeloproliferative neoplasms, Version 2.2018, Available at: . Accessed: February 14, 2018.
    1. Aaronson NK, Ahmedzai S, Bergman B, et al. The European Organization for Research and Treatment of Cancer QLQ-C30: a quality-of-life instrument for use in international clinical trials in oncology. J Natl Cancer Inst 1993; 85:365–76.
    1. Reilly MC, Zbrozek AS, Dukes EM. The validity and reproducibility of a work productivity and activity impairment instrument. Pharmacoeconomics 1993; 4:353–65.
    1. Charlson ME, Pompei P, Ales KL, MacKenzie CR. A new method of classifying prognostic comorbidity in longitudinal studies: development and validation. J Chronic Dis 1987; 40:373–83.

Source: PubMed

Sponsors en medewerkers

Medische omstandigheden

Geneesmiddelinterventies

3
Abonneren