HOUSTON — A retrospective study presented at the Society of Hematologic Oncology (SOHO) 2025 suggested that glucagon-like peptide-1 receptor agonists (GLP-1s) such as semaglutide (Ozempic/Wegovy) — widely used for diabetes and weight loss — might also have a role in polycythemia vera (PV), a myeloproliferative disorder characterized by overproduction of red blood cells.
After thorough 1:1 propensity score matching, investigators found that across 4119 matched pairs of patients with PV, those who started GLP-1s vs those who did not had lower odds of all-cause mortality (odds ratio [OR], 0.490), myelofibrosis progression (OR, 0.548), and venous thromboembolism (VTE; OR, 0.705) over the next 3 years.
GLP-1 users also had lower odds of all-cause hospitalizations (OR, 0.688), ICU admissions (OR, 0.523), acute kidney injury (OR, 0.679), and ischemic stroke/transient ischemic attack (OR, 0.831). The findings were all statistically significant.
“If this were a drug developed for PV, it would be considered very successful. You’d see booths” at the meeting promoting it, said an audience member after lead investigator Asfand Yar Cheema, MD, internal medicine resident at the Cleveland Clinic in Cleveland, Ohio, presented the findings.
“The results are very promising,” Cheema told Medscape Medical News, and raised the possibility of incorporating GLP-1s into PV care if they pan out in randomized trials.
If the drugs truly turn out to help in PV, it may be because they dial down chronic inflammation, which could make cancer-causing mutations in key cellular pathways less likely. Lab studies in various cell lines support the idea.
It’s also possible, however, that patients on GLP-1s simply lost more weight than their peers, said Neil Iyengar, MD, breast cancer medical oncologist and GLP-1 researcher at the Emory University, Atlanta.
“Patients with polycythemia vera are more likely to be obese, and obesity is associated with worse outcomes. Endpoints in the study — all-cause mortality, VTE, and healthcare utilization — are known to improve with obesity treatment, so these findings are not surprising. It’s not clear whether they are specific to an effect in PV or related to the anti-obesity benefits” of GLP-1s, he said.
The data in Cheema’s study did not capture whether patients on GLP-1 lost more weight than their matched control participants.
Iyengar noted the drugs have also shown benefit in patients with breast and other cancers, but again it’s unclear if it’s due to specific anticancer effects or simply weight loss. There’s also emerging data that they may help prevent obesity-related tumors.
As for blood cancers, a study earlier this year found that GLP-1 use vs metformin or insulin was associated with a lower risk for hematologic malignancies among patients with type 2 diabetes.
Propensity score matching in Cheema’s study included demographics and baseline cardiovascular, renal, and hepatic comorbidities plus obesity, PV medications, A1c, and cholesterol levels, among other factors.
Over half of the GLP-1 patients were on semaglutide, with the rest on other GLP-1s. Patients were on treatment for an average of 297 days.
Data came from the TriNetX Analytics Network database, which compiles de-identified electronic health records across more than 30 countries, including the US.
Among the study’s limits, patients were treated at 147 institutions. Variations in PV care may have affected the results, Cheema noted. He and his team next plan to study the effect of GLP-1s on PV clonal evolution.
This study did not receive any funding. Cheema didn’t have any disclosures. Iyengar reported being an advisor and/or researcher for several companies including AstraZeneca, Roche, Novartis, Pfizer, and SynDevRx.
M. Alexander Otto is a physician assistant with a master’s degree in medical science and a journalism degree from Newhouse. He is an award-winning medical journalist who worked for several major news outlets before joining Medscape. Alex is also an MIT Knight Science Journalism fellow. Email: aotto@mdedge.com.