GLP-1 Therapy Linked to Improved Outcomes in Polycythemia Vera

Treatment with glucagon-like peptide-1 receptor agonists (GLP-1a) was associated with statistically significant reductions in all-cause mortality, progression to myelofibrosis, and venous thromboembolism, as well as lower rates of hospitalization, intensive care unit (ICU) admission and acute kidney injury among patients with polycythemia vera, a type of blood cancer. It was also associated with reduced rates of ischemic stroke or transient ischemic attack, according to findings from a large-scale analysis presented at the 2025 SOHO Annual Meeting.

“Every parameter, both for primary and secondary outcomes, was statistically significantly decreased in patients who were taking a GLP-1a versus those who were not,” presenting author Dr. Asfand Yar Cheema, stated in the presentation.

He is a hematology/oncology resident physician at the Cleveland Clinic in Columbus, Ohio.

Among patients with polycythemia vera, those receiving GLP-1a experienced lower rates of several side effects compared with non-users. Acute kidney injury occurred in 11.72% of GLP-1a users versus 16.37% of non-users. Progression to myelofibrosis was 1.70% in users, compared with 3.06% in non-users.

Venous thromboembolism affected 8.33% of patients versus 11.41% in non-users and ICU admissions occurred in 7.45% of users versus 13.35% of non-users; all-cause hospitalizations were 44.82% versus 54.14%, respectively. Mortality was 4.47% among users compared with 8.72% in non-users. Rates of ischemic stroke or transient ischemic attack were 7.45% for patients versus 8.84% for non-users.

Polycythemia Vera Overview and Emerging Role of GLP-1a

Polycythemia vera is a chronic myeloproliferative neoplasm, with approximately 95% of patients harboring the JAK2 V617F mutation. Key complications include venous thromboembolism, which occurs in approximately 34% to 41% of patients, progression to myelofibrosis in 10% to 15% and leukemic transformation in 3% to 4%.

GLP-1a, primarily used for glycemic control and weight management, have emerging evidence suggesting they may reduce the risk of myelodysplastic syndromes and myeloproliferative neoplasms in patients with type 2 diabetes mellitus or autoimmune disorders. Preclinical studies indicate these therapies possess antineoplastic and anti-inflammatory activity, producing cytostatic effects and modulation of the JAK/STAT pathway.

Building on the preclinical rationale, investigators aimed to evaluate the association between GLP-1a therapy and key clinical outcomes in patients with polycythemia vera using a large global database. The analysis focused on primary outcomes of all-cause mortality, progression to myelofibrosis and venous thromboembolism. Secondary outcomes included all-cause hospitalizations, intensive care unit admissions, acute kidney injury and transient ischemic attack.

The study was conducted using data from the TriNetX Analytics Network during 2010 to 2022, encompassing 147 healthcare organizations. The population included 5,291 patients with polycythemia vera receiving GLP-1a and 79,027 patients who were not using these therapies.

Patients were followed for three years from the index event, defined as the time of polycythemia vera diagnosis while receiving GLP-1a therapy. The mean duration of therapy among patients was approximately 298 days, with a standard deviation of approximately 162 days.

Study Limitations and Future Research Directions

Overall, researchers emphasized that the use of GLP-1a led to a therapeutic benefit among patients. Additionally, therapy was linked to reduced healthcare utilization, including fewer hospitalizations and intensive care unit admissions, as well as lower rates of acute kidney injury and transient ischemic attack.

“Even when we looked specifically at patients with type 2 diabetes, GLP-1a showed very beneficial results,” Cheema highlighted.

However, the study authors highlight important limitations. Its observational, retrospective design precludes conclusions about causality, and the TriNetX database offers limited clinical granularity. Outcomes may be under-ascertained when patients receive care outside participating institutions, and there is the potential for misclassification or miscoding of diagnoses.

Despite these limitations, the findings suggest a potential therapeutic benefit of GLP-1a in high-risk polycythemia vera patients. Randomized controlled trials are warranted to confirm both the safety and efficacy of therapy in this population.

“Our study suggests a therapeutic benefit of GLP-1a in patients with polycythemia vera… although randomized controlled trials are needed to confirm safety and efficacy in this high-risk population,” he concludes.

Reference

1. “Glucagon-Like Peptide-1 Agonists and Clinical Outcomes in Polycythemia Vera: A Large-Scale Propensity-Matched Cohort Study” by Asfand Yar Cheema. Presented at: 2025 SOHO Annual Congress; Sept. 3-6, 2025; Houston, TX.

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