New Hope for HHT Patients: Pomalidomide Shows Promise in Treating Rare Bleeding Disorder
Researchers have found that pomalidomide, a drug used for bone marrow cancer, is effective in treating hereditary hemorrhagic telangiectasia (HHT), a rare bleeding disorder. The trial, funded by the National Institutes of Health, showed significant improvements in patients, including reduced nosebleeds and fewer blood transfusions. Dr. Andrei Kindzelski from NIH highlighted the success, noting the lack of reliable treatments for HHT before this discovery.
Understanding HHT
HHT, also known as Osler-Weber-Rendu Syndrome, causes blood vessels to form abnormally, leading to excessive bleeding. Current treatments involve closing malformed vessels or using temporary medications, but no FDA-approved long-term solutions exist.
The Clinical Trial
Led by Dr. Keith McCrae at the Cleveland Clinic, the trial involved 144 adults with HHT. Participants received either pomalidomide or a placebo. Results showed pomalidomide’s effectiveness, leading to the trial’s early termination. The drug may help normalize blood vessel structure, reducing fragility.
Future Implications
Dr. Kindzelski noted the potential for pomalidomide to help severe HHT cases, where malformed vessels in organs can cause serious complications. Some patients experienced long-term benefits, suggesting the drug’s promise for ongoing treatment.
Doubts Revealed
HHT -: HHT stands for Hereditary Hemorrhagic Telangiectasia. It is a rare genetic disorder that causes abnormal blood vessels, leading to frequent nosebleeds and other bleeding issues.
Pomalidomide -: Pomalidomide is a medicine originally used to treat a type of cancer called bone marrow cancer. It is now being tested to see if it can help people with HHT.
NIH -: NIH stands for National Institutes of Health. It is a part of the U.S. government that supports medical research to improve health.
Blood transfusions -: Blood transfusions are a medical procedure where a person receives blood through a vein. It is often done when someone loses a lot of blood or has a blood disorder.
Dr. Keith McCrae -: Dr. Keith McCrae is a doctor and researcher who led the study on using pomalidomide to treat HHT. He works to find better treatments for people with this disorder.
