Burden of thromboembolism, disease progression, and phlebotomy among polycythemia vera patients in the United States: a systematic literature review.

INTRODUCTION

Patients with polycythemia vera (PV) can experience thromboembolism (TE) and progress to myelofibrosis, acute myeloid leukemia, or myelodysplastic syndromes. Treatments for PV, particularly phlebotomy, may be burdensome and reduce patient quality of life and productivity. A systematic review was conducted to characterize the burden of TEs, disease progression, and phlebotomy among PV patients in the United States.

METHODS

Embase, MEDLINE and MEDLINE In-Process, PsycInfo, Cochrane Database of Systematic Reviews, and conference proceedings were searched for relevant studies published from 2014 (2023 for conference abstracts) through June 2025. Evidence was synthesized narratively.

RESULTS

Thirty-five unique studies were identified. Patients with PV experienced 1.4 to 6.5 TEs per 100 patients per year; likely risk factors for TEs included prior TEs and elevated hematocrit or hemoglobin levels. PV patients with TEs experienced higher patient healthcare costs, resource utilization, and mortality risk compared with PV patients without TEs. Disease progression was most often due to myelofibrosis (2.3%-19.9% of patients). Treatment with phlebotomy was linked with reduced quality of life and lost productivity.

CONCLUSIONS

Some patients with PV may experience a substantial burden, including risk of TEs, impacts to quality of life and productivity, and disease progression, despite treatment with the current standard of care.

REGISTRATION: This systematic review was prospectively registered with PROSPERO (CRD420251078378), with the authors confirming that only pilot work took place before any data collection occurred (including searching, screening, or data extraction).