P1692: unmet needs in patients with “low risk” polycythemia vera (pv): symptom burden and quality of life

Topic: 35. Quality of life and palliative care 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). Aims: The study, therefore, aimed to explore symptom burden and QoL in “low risk” PV patients and to compare such aspects with that in “high risk” PV. Methods: Patients with PV were selected from a cohort of participated in a cross-sectional nationwide survey MPN-QoL-2020 carried out in September-December 2020 in different areas of Russia. Patients aged ≥18 years with a confirmed diagnosis of PV and treated on the in-patient or outpatient basis were included in the analysis. For symptom and QoL assessment MPN10 and HM-PRO were used, respectively. The MPN10 assesses 10 of the most clinically relevant symptoms, including fatigue and generates a Total Symptom Score (TSS). The HM-PRO is a specific QoL questionnaire for patients with hematologic malignancies developed for the use in clinical practice. It consists of two scales: Part A measuring the ‘impact on patients’ QoL’; Part B – ‘signs and symptoms’ (S&S) experienced by the patients. Part A has 4 domains: physical behavior (PB), social well-being (SW), emotional behavior (EB), eating and drinking habits (ED). Higher scores indicate greater impact. Exploratory analyses were carried out applying descriptive statistics, χ2 test and Mann-Whitney U test. Results: Altogether 265 PV patients were included in the analysis: 95 patients were identified as “low risk” (mean age±SD – 48.8±11.6 yrs; 49.5% males) and 170 patients as “high risk” PV (mean age – 62.9±12.3 yrs; 45.3% males). In the “low risk” group, 69.5% received cytoreductive treatments (CT) – hydrea (64%), interferons (30%), ruxolitinib (6%); in the high risk group, 86.5% received CT – hydrea (79%), interferons (15%), ruxolitinib (6%). Among “low risk” PV, 94.7% reported at least 1 PV-related symptom. The most frequently reported symptoms of MPN10 were inactivity (83%), fatigue (81%) and itching (65%). Symptoms with the highest reported mean severity scores were fatigue (3.3±2.6), inactivity (3.3±2.6), problems concentrating (2.8±2.9) and itching (2.6±2.9). More than half of “low risk” patients (61.5%) experienced moderate-to-severe symptoms (4-10 scores). Symptom burden according to MPN10 TSS was similar to “high risk” patients: 20.4±16.5 vs 24.6±18.4 (ns). Prevalence of moderate-to-severe symptoms did not differ significantly between “low risk” (61.5%) and “high risk” (71.7%) PV (ns). In “low risk” PV, the highest impact on QoL by HM-PRO (Part A) was for EB (Table). According to HM-PRO Part A and S&S, 26.3% and 44.2% patients exhibited moderate/very large/extremely large effect on QoL and S&S, respectively (Table). In “high risk” PV these proportions were 38.2% and 52.3%, respectively (ns). QoL impact and S&S were higher in “high risk” than in “low risk” PV (p<0.05), Table. Summary/Conclusion: In conclusion, the vast majority of patients with “low risk” PV reported high PV-related symptoms/ symptom burden and impact on QoL. Further prospective studies are worthwhile to explore the determinants of symptom burden in “low risk” PV. Furthermore, the results suggest that risk profiling may not capture all aspects of disease burden in PV and this highlights the importance of using patient-reported outcomes, namely MPN10 and HM-PRO, to identify unmet patients’ needs, especially in “low risk” PV.Keywords: Risk factor, Quality of life, Polycythemia vera