UVM Cancer Center Clinical Trial Showed Drug’s Effectiveness
Marie A. LaCourse of Newark, Vt. participated in a pragmatic clinical trial at the University of Vermont Cancer Center demonstrating that a pill was as effective as an injected blood thinner in preventing blood clots from returning in cancer patients.
For the five to 25 percent of cancer patients who develop a potentially fatal condition called venous thrombosis, which causes clots to form in the blood that can lodge in the lungs or other vital organs, the anxiety of a cancer diagnosis comes with an unpleasant added chore — the need to inject themselves once or twice a day with a blood thinner called heparin.
Given the seriousness of cancer-related venous thrombosis — the recurrence of a clot after the first can be lethal — the injection regimen is an absolute necessity.
But the discomfort the life-saving treatment causes should not be underestimated, says Chris Holmes, M.D., Ph.D., associate professor of hematology/oncology, and a hematologist who specializes in cancer at the University of Vermont Cancer Center.
“You have to inject yourself in your abdomen or your leg, and the more you weigh, the more medicine you need,” Holmes said. “The injection can create large bruises or knots you have to avoid next time. If you hit an artery and bleed, you need to come to the clinic or hospital. And for 10 percent to 20 percent of patients, the injection hurts; they can feel the medication spread.”
Pill as effective as shot
Thanks to the positive results generated by a new clinical trial that the UVM Cancer Center and Holmes played a role in, many more patients will no longer need to endure this distress.
The trial demonstrated conclusively that an orally administered pill — a DOAC, or direct oral anticoagulant — was as effective in preventing the recurrence of blood clots as injected heparin and did not increase the risk of bleeding.
“It’s a huge improvement in quality of life,” said Holmes, both for patients and for caregivers like spouses and children, who are often saddled with the job of injecting their loved one, Holmes said.
Marie A. LaCourse was one of these patients. A school bus driver for 30 years in the Northeast Kingdom, LaCourse was diagnosed five years ago with a brain tumor. With surgery, the tumor was removed, but six months later she was hospitalized again with a potentially life-threatening blood clot in her lungs. She started treatment with injectable blood thinners.
“My husband did the shot, and I held the skin. He was a very kind person,” recalls LaCourse. It was her doctor, Alissa Thomas, M.D., associate professor of neurological sciences and a neuro-oncologist at the UVM Cancer Center, who told her about the clinical trial. “I was skeptical at first, but the pills were much easier,” said LaCourse. “I am thankful to Dr. Thomas for making my life easier. Hopefully someone will learn from my experience and seek out support.”
The results of the trial were published in The Journal of the American Medical Association, a top-five medical journal.
“Pragmatic” Clinical Trial Boosts Confidence in, Use of the New Drug
The research paper summarized the results of what is called a pragmatic clinical trial. Unlike traditional clinical trials, which are small, rigorously controlled in a medical setting, and open only to a target group of patients with a set of defined characteristics, pragmatic trials are large, take place outside the hospital, and are open to a broad swath of patients.
While the small, controlled trials are the vehicle by which drugs obtain FDA approval — the DOACs in the pragmatic trial were approved in this manner several years ago — large pragmatic trials often lead to a drug becoming more widely prescribed by medical practitioners and widely covered by insurance companies.
“Pragmatic trials boost confidence in a drug because it’s tested it in a real world setting,” said Holmes.
The trials “have the advantage that they’re more relevant to broad groups of patients, so new medications can be rapidly implemented and incorporated into clinical practice,” said Randall Holcombe, M.D., M.B.A., professor of medicine, chief of the hematology/oncology division, and director of the UVM Cancer Center.
To generate a large sample of patients, 67 institutions recruited participants for the clinical trial. UVM enrolled 22 patients in the trial. Holmes played a role not just in supplying data from the UVM participants, but also in reviewing and analyzing it, and in helping write the final paper.
Why Clots Develop, Who’s Most Vulnerable
Cancer patients are prone to developing blood clots because the disease itself can make blood cells “sticky” and more inclined to clot, Holmes said. In addition, chemotherapy treatments can promote increased clotting, she said.
The likelihood of developing clots is dependent on the cancer type; it occurs most frequently in pancreatic, lung, brain, colorectal and blood cancers.
Older and more immobile patients are also more vulnerable.
About the University of Vermont Cancer Center: The University of Vermont Cancer Center is Vermont’s only not-for-profit comprehensive clinical and research cancer center. Founded in 1974, the organization is located within the University of Vermont Larner College of Medicine and enjoys a clinical partnership with the University of Vermont Medical Center. Drawing on the expertise of more than 170 research and clinical members, the center works to reduce the burden of cancer in Vermont, northeastern New York and across northern New England, through research, outstanding clinical care, community outreach, and education.