Abstract Topic: 16. Myeloproliferative neoplasms - Clinical
Background: The main symptoms of polycythemia vera (PV) include fatigue (general malaise), pruritus, night sweats, decreased concentration and motivation, and splenomegaly-related symptoms such as early satiety and abdominal discomfort and/or pain. PV is generally progressive, and the physical symptoms worsen with disease progression, adversely affecting patients’ daily living and employment. Several studies have examined patients’ perceptions of which PV symptoms have the greatest impact on their daily living and have compared symptom and treatment perceptions between patients and physicians. However, these perceptions have not yet been fully evaluated in Japan.
Aims: The primary aim of this study was to clarify which symptoms patients with PV perceive as burdensome, and thereby determine how PV symptom burden affects their daily living. The secondary aim was to understand the gaps between patients and physicians in some of their perceptions, such as symptom and the treatment goals.
Methods: This was a cross-sectional survey of Japanese patients with PV and their physicians. Participants were recruited from March to July 2022. The patient and physician questionnaires comprised 34 and 29 questions (partially multiple choice), respectively. Questions regarding PV symptoms were formulated based on the MPN Landmark Survey and the MPN Symptom Assessment Form. The impact of PV on daily living was assessed on a scale of five possible responses: no impact, little impact, somewhat impact, impact, and large impact.
Results: A total of 265 patients and 151 physicians from 112 facilities were included in the analysis set. Regarding the patients’ characteristics, 148 (55.8%) were male. The mean age was 67.3 years; 205 patients (77.4%) were aged ≥60 years, and 58 patients (21.9%) were aged <60 years. Regarding the physician characteristics, 116 (76.8%) were male, and the mean age was 48.5 years.
The PV symptoms that patients most wanted to have resolved were pruritus (including after a hot bath or shower) (n=36, 13.6%), and fatigue (n=29, 10.9%). Meanwhile, the PV symptoms that physicians perceived as most important to resolve were fatigue (n=51, 33.8%), and headache (n=30, 19.9%).
The impact of PV on daily living based on five items, namely family life, social life, work, leisure activities, and sex life, were assessed (Figure). More than half of the patients answered “no impact” for each of the items. By contrast, the percentage of physicians who reported “no impact” on any of the items was around 10%, which was notably lower than the results for patients. In the subgroup analysis by patient age, the percentage of patients who reported a greater impact (“large impact” or “impact”) for all five items was higher in the subgroup aged <60 years than in the subgroup aged ≥60 years.
Regarding treatment goals, patients placed the highest value on “slow disease progression”, as well as “prevention of thrombotic events”. By contrast, physicians placed the highest value on “prevention of thrombotic events”. “Slow disease progression” was selected by 65 (24.5%) of patients versus only 4 (2.6%) of physicians.
Summary/Conclusion: For more than half of the patients, PV had little impact on their daily living. However, a higher percentage of patients aged <60 years reported a greater impact compared with patients aged ≥60 years. It should be noted that discrepancies exist between patients and physicians in their perception of the symptoms, and treatment goals.
Keywords: Patient, Polycythemia vera