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Keith McCrae, MD

Professor, Department of Molecular Medicine, Cleveland Clinic Lerner College of Medicine of the �Ǹ���� School of Medicine
Member, Case Comprehensive Cancer Center
Director of classical hematology, Cleveland Clinic Taussig Cancer Institute
Director of physician-scientist development, Cleveland Clinic Lerner Research Institute
Member, American Society of Hematology (ASH)
Editor-in-Chief of Blood Vessels, Thrombosis and Hemostasis, an American Society of Hematology publication
Area of focus: translational research in vascular biology, including studies on endothelial cells, platelets and other blood cells.

When a patient presented with challenging nosebleeds and severe gastrointestinal bleeding—hallmarks of Hereditary Hemorrhagic Telangiectasia (HHT), a rare genetic disorder affecting blood vessel formation—hematologist Keith McCrae was determined to find an effective treatment.

In patients with HHT, blood vessels develop abnormally and are prone to leaking. The disorder causes excessive nosebleeds and life-threatening complications in multiple organs.

McCrae’s patient had limited options and had been advised to undergo bowel surgery, which would have meant he’d depend on intravenous nutrition. Instead, McCrae treated this patient with a cancer drug that showed promising results in patients with similar symptoms. 

The approach was a success. His patient’s bleeding stopped within weeks, McCrae  published by The Clinical Research Forum, a nonprofit association supporting academic health centers and researchers. The organization—which includes CWRU’s Clinical and Translational Science Collaborative—recently honored McCrae with a Top 10 Clinical Research Achievement Award, in recognition of his New England Journal of Medicine article, “.”

“The ideas for this study were partly conceived when I was a Case Western Reserve faculty member treating many patients with rare bleeding disorders that were not well understood,” said McCrae, who’s been interested in studying blood clotting and vascular function since medical school. “My research eventually led to testing the use of pomalidomide.”

McCrae received funding from the National Institutes of Health to develop a national multi-site clinical trial to test the efficacy of pomalidomide, a drug typically used to treat a type of bone marrow cancer. Pomalidomide is a similar drug to the one that helped his patient, but it had fewer side effects than for the large-scale trial, as explained in a .

Study participants had a significant decrease in severe nosebleeds, required fewer blood and iron transfusions and had an improved quality of life—providing evidence of a safe and effective drug for HHT patients who struggle with nosebleeds. 

Stopped early because of its success, the study was the first positive systemic trial for an HHT treatment, and the studies suggest that pomalidomide may be a useful therapy for patients with HHT.

“My advice to those early in their career and anyone in the field is do not ignore a fascinating clinical outcome just because it only occurred in one patient you may have encountered,” McCrae said.