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 worldwide. The trial, led by Keith McCrae, M.D.
, of Cleveland Clinic, was stopped early because of these successful findings, and has been published in the New England Journal of Medicine . The impetus for this trial was a single patient. About 15 years ago, Dr.
McCrae, director of Benign Hematology at Cleveland Clinic, and the study's senior author, saw a patient with HHT symptoms. There was little information about the disease at the time, according to Dr. McCrae, a hematologist and scientist.
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, affect quality of life, and can lead to anemia and other life-threatening conditions. There are no FDA-approved medications for long-term management of HHT. The patient, who was in his 50s at the time, was experiencing nosebleeds and severe GI bleeding.
He required several blood transfusions and multiple doses of concentrated blood plasma clotting factors every week. He had been told that his only option was surgery to remove his diseased bowel, which would have significantly affect.