Clinical trial demonstrates success in treating rare blood disorder

Medical Xpress / Cleveland Clinic / New England Journal of Medicine ^ | Sept. 18, 2024 | Keith McCrae, M.D. et al

[ConservativeMind]

A clinical trial has demonstrated that the cancer drug pomalidomide is safe and effective in treating hereditary hemorrhagic telangiectasia (HHT), a rare bleeding disorder that impacts more than one in 5,000 people.

The trial was stopped early because of these successful findings.

HHT is a condition in which blood vessels become unusually tangled and twisted. This can lead to excessive nosebleeds that hallmark the disease. HHT also causes bleeding in the digestive tract and can produce serious complications in the lungs, liver and brain. Bleeding episodes worsen with age. There are no FDA-approved medications for long-term management of HHT.

Dr. McCrae began searching for new options and found that the cancer drug thalidomide had shown positive outcomes in a few patients with similar symptoms.

Thalidomide primarily treats multiple myeloma, but can have serious side effects, so instead of pursuing large-scale research with thalidomide, Dr. McCrae used a drug with a similar chemical structure called pomalidomide, an FDA-approved drug for the treatment of bone marrow cancer.

All participants had moderate to severe nosebleeds requiring iron infusions or blood transfusions.

Researchers observed that patients with HHT taking pomalidomide saw a marked decrease in nosebleed severity, required fewer blood transfusions and iron infusions and experienced an improved quality of life.

Researchers speculate that pomalidomide works by blocking the growth of abnormal blood vessels.

Though researchers did not follow participants after the trial ended, Dr. McCrae said some of his patients who were enrolled in the study have gone up to six months without nosebleeds recurring, even though they had stopped the medication. This suggested that the drug may have promise as a long-term or intermittent treatment, he said.

[ConservativeMind]

Pomalidomide appeared to be helpful at least six months after taking it.

[ConservativeMind]

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