Burden of illness in polycythemia vera: A retrospective medical chart audit study.
Authors: Ahmed Tasfia , Hobbs Gabriela S. , Sirignano David S. , Foster Judy , Sosa Adan , Chien Hung-Lun , Flynn Megan , Castro Claudia , Kulkarni Aishwarya , Skaar Jeffrey R. 
Published: June 2024

e18530

Background: Polycythemia Vera (PV) is a rare, chronic, and life-threatening myeloproliferative neoplasm (MPN) associated with significant clinical, economic, and humanistic burden. This study evaluates the real-world burden of illness and treatment for PV, including patient management and treatment effectiveness, through the evaluation of patient characteristics, disease progression, and clinical treatment outcomes. Methods: A retrospective medical chart review was conducted in Oct-Dec 2023 among US healthcare providers (HCPs) managing adult patients with a confirmed diagnosis of PV in the prior 12 months. At least 30 charts were solicited for patients receiving phlebotomy alone (PLB), and each of the following cytoreductive therapies (CT) with or without concomitant PLB: hydroxyurea (HU), ruxolitinib (RUX), peginterferon alfa-2a (PEG), and ropeginterferon alfa-2b (ROPEG). Data were collected at three points in time, initial presentation of PV, diagnosis, and at the most recent HCP visit; additionally, HCP rationale on treatment decision-making was captured. Results: A total of 125 HCPs from academic (30%), community (28%), and private (33%) practices abstracted data from 420 unique charts. The mean patient age was 61.5±11 years and 266 (63%) patients were identified as high risk (≥60 years or history of thrombosis), including patients who were on PLB alone (56). Among patients who received CT first line (1L) were: (HU:83, RUX: 14, PEG: 4, ROPEG: 3). Among patients currently on PLB alone (n=103), the top three reasons to initiate CT would be worsening symptoms (32%), increased PLB frequency (29%), or difficulty to control hematocrit (24%). Most patients (n=317) had experience on ≥1 CT (HU: 165, RUX: 115, PEG: 44, ROPEG: 45). Across therapies, HCPs observed a reduction in severe symptoms from initial presentation to the most recent visit (HU: 43% (7 to 4 symptom reduction) , RUX: 45% (11 to 6), PEG: 33% (12 to 8), ROPEG: 75% (12 to 3); common persisting symptoms across all treatments were night sweats and burning/tingling in periphery. HCP observed severe adverse events (SAEs) across all treatments (HU: 6%, RUX: 5%, PEG: 11%, ROPEG: 11%); of the 19 patients experiencing AEs on RUX discontinued due to neutropenia (5%) and GI bleeding (5%), similarly, 79 patients experiencing AEs on HU discontinued due to liver enzyme elevation (3%) and thrombocytopenia (3%), 24 patients and 18 patients experiencing AEs on PEG and ROPEG discontinued due to dizziness (8%), unusual bleeding (4%), and increased blood pressure (6%), skin conditions (6%) respectively. Conclusions: CT, including disease modifying therapeutic options beyond HU, demonstrated efficacy in lowering the symptom burden of PV, in which ropeginterferon demonstrated the most favorable results. The persistence of severe symptoms and AEs demonstrates that some patients have an unmet need for more efficacious and tolerable treatments and management strategies.