Clinical Summary:

• Design/Population: This retrospective TriNetX analysis compared propensity score-matched adults with essential thrombocythemia who received GLP-1 receptor agonists with patients who were not exposed to GLP-1 therapy, evaluating clinical outcomes over 5 years. 
• Key Outcomes: GLP-1 receptor agonist use was associated with lower mortality and reduced risks of sepsis, atrial fibrillation, hospitalization, transfusion requirements, and venous thromboembolism. Rates of coronary artery disease, bleeding, and stroke were not significantly different between groups. 
• Clinical Relevance: These findings suggest that GLP-1 receptor agonists may provide benefits beyond their established metabolic effects in patients with essential thrombocythemia, particularly for survival and thrombotic outcomes, although prospective studies are needed before establishing a treatment role in essential thrombocythemia. 

Results from a large retrospective cohort study demonstrated that glucagon-like peptide 1(GLP-1) receptor agonist use was associated with improved survival and lower rates of severe thrombotic, cardiovascular, and infectious complications among patients with essential thrombocythemia.

These results were presented by Nneoma Ubah, MD, Montefiore St Luke Cornwall Hospital, Newburgh, New York, at the Society of Hematological Oncology (SOHO) Annual Meeting in Houston, Texas.

Investigators conducted this analysis using the TriNetX US Collaborative Network. Adult patients with essential thrombocythemia were identified using International Classification of Diseases, 10th Revision and International Classification of Diseases for Oncology, 3rd Edition codes. Patients with polycythemia vera, primary myelofibrosis, or acute myeloid leukemia were excluded. 

Patients receiving GLP-1 receptor agonists were compared with those who had no GLP-1 exposure. Propensity score matching was performed according to demographics, cardiovascular comorbidities, diabetes, chronic kidney disease, obesity, atrial fibrillation, thrombotic history, and hemoglobin, resulting in 16,263 patients in each cohort. 

Clinical outcomes were evaluated over 5 years and included mortality, sepsis, atrial fibrillation, venous thromboembolism, bleeding, stroke, transfusion requirements, hospitalization, coronary artery disease, and transformation to acute myeloid leukemia.

Mortality was significantly lower among patients receiving GLP-1 agonists, occurring in 8.3% compared with 13% of patients without GLP-1 exposure (hazard ratio [HR], 0.6; 95% confidence interval [CI], 0.56 to 0.64; P < .001). The 5-year survival probability was 84.2% and 77.7%, respectively.

GLP-1 receptor agonist use was also associated with a lower incidence of venous thromboembolism, which occurred in 2.8% of patients receiving GLP-1 therapy compared with 4% of patients without exposure (P < .001). 

Additional differences favored GLP-1 receptor agonist use. Sepsis occurred in 6.2% versus 9% of patients (P < .001), while atrial fibrillation occurred in 3.4% versus 4.2% of patients (P = .001). 

Patients receiving GLP-1 receptor agonists were also less likely to require transfusions, with rates of  3.8% compared with 5.9% among patients without GLP-1 exposure (P < .001). Hospitalization occurred in 11.1% and 16.6% of patients, respectively (P < .001). 

In contrast, rates of coronary artery disease, bleeding, and stroke were not significantly different between the matched cohorts. No acute myeloid leukemia transformation was observed in either group during the evaluation period. 

Although propensity score matching accounted for several baseline cardiovascular and metabolic factors, the retrospective observational design establishes an association rather than a causal effect of GLP-1 receptor agonist therapy. Prospective investigation will be necessary to determine whether GLP-1 receptor agonists directly influence thrombotic or cardiovascular risk in essential thrombocythemia.

The investigators concluded that GLP-1 receptor agonist exposure was associated with improved survival and reductions in several clinically important complications, raising the possibility of a protective role for these agents in patients with essential thrombocythemia.

Source:

Ubah N, Ugwu C, Omenuko N, et al. Impact of GLP-1 agonists on clinical outcomes in patients with essential thrombocythemia: A propensity-matched analysis. Presented at the SOHO Annual Meeting; September 9-12, 2026; Houston, Texas. Abstract MPN-1689.