Skip to main content
Home » Rare Diseases » A Rare Blood Cancer May Lead to Heart Attack and Stroke
Rare Diseases

A Rare Blood Cancer May Lead to Heart Attack and Stroke

pv-rare diseases-blood cancer-cancer
pv-rare diseases-blood cancer-cancer

A cancer that thickens the blood and causes fatigue can also lead to heart attack and stroke.

Polycythemia vera (PV) is a chronic cancer that leads to overproduction of red blood cells, increasing “stickiness” of blood. People with PV experience a range of symptoms, some of which may be dismissed as less serious. These include visual disturbances, shortness of breath, severe itching and poor concentration.

But the excess number of red blood cells slows blood flow, which puts people with PV at risk for additional serious health threats, including blood clots and cardiovascular events like heart attacks, strokes and mini-strokes.

If diagnosed, people with PV can regularly undergo a procedure called phlebotomy. Phlebotomy removes blood from the body using the same procedure as donating a unit of blood.

“The established goal of phlebotomy therapy is to maintain red blood cell levels, as measured by hematocrit, below 45%,” says Dr. Ronald Hoffman, Albert A. and Vera G. List professor of medicine and director of the Myeloproliferative Disorders Research Program at the Icahn School of Medicine at Mount Sinai in New York, New York. “Research shows phlebotomy is an effective treatment, but frequently the target hematocrit level is not achieved between physician visits, leaving the patient at risk for developing clots.”


Patients with poorly controlled PV can develop scarring of their marrow known as myelofibrosis and can sometimes develop leukemia.

“PV is one of those blood cancers that you don’t hear about as much, but it is a serious disease. It’s important that patients get diagnosed and understand their treatment options,” Dr. Hoffman says. “In addition to phlebotomy, there are a number of approved medicines that can be used to control this condition, and experimental therapeutics are also being evaluated.”

An example is rusfertide. An investigational medicine, it is still being studied by doctors and is not approved by regulatory authorities like the US Food and Drug Administration (FDA) or the European Medicines Agency (EMA).

Rusfertide is currently being studied to evaluate its safety and ability to control the production of red blood cells and to keep hematocrit levels within the recommended range, potentially reducing the need for phlebotomies, its ability to impact symptoms as well as if patients experience fewer blood clots or cardiovascular complications.

“We hope that by advancing research and development of drug candidates like rusfertide, we may be able to potentially add to available treatment options for PV and other cancer and rare diseases,” says Dr. Molina, a physician-scientist specializing in the development of new treatments for rare and more prevalent diseases at Protagonist Therapeutics.

This article has been paid for by Protagonist Therapeutics.

Next article