Hereditary haemorrhagic telangiectasia (HHT) can be treated with pomalidomide, according to the findings of a groundbreaking study.
A US study was halted early because of the success of the research, which involved 144 patients at 11 medical centres, starting in 2019. It makes it the first drug successfully tested in a trial as a treatment for the disorder.
The findings were reported in the New England Journal of Medicine last week. Researchers reported a “marked” decrease in nosebleed severity and reductions in blood transfusions and iron infusions.
The study was conceived 15 years ago when study leader Dr Keith McRae, of the Cleveland Clinic, Ohio, tried treating a patient with thalidomide. The patient was suffering nosebleeds and severe gastrointestinal bleeding – and there was little information available about the disease.
Dr McRae found cases of the drug being used to treat similar symptoms and decided to try thalidomide on the patient. Pomalidomide was chosen for the trial as a safer drug.
Dr McRae said: “I had rarely seen anything quite like that in my clinical experience, and I thought, it is important that we study this.
“I'm optimistic that learning more about the mechanisms of how this treatment works will make a great impact in treatment of hereditary haemorrhagic telangiectasia haematology and our understanding of vascular malformations.”
Dr Andrei Kindzelski, of the Division of Blood Diseases and Resources at the US National Heart, Lung, and Blood Institute, said: “Finding a therapeutic agent that works in a rare disorder is highly uncommon, so this is a real success story. To date, there has been no positive trial of a therapeutic for treating HHT.”
Source:
Al-Samkari H, Kasthuri RS, Iyer VN, Pishko AM, Decker JE, Weiss CR, Whitehead KJ, Conrad MB, Zumberg MS, Zhou JY, Parambil J, Marsh D, Clancy M, Bradley L, Wisniewski L, Carper BA, Thomas SM, McCrae KR. (2024) “Pomalidomide for Epistaxis in Hereditary Hemorrhagic Telangiectasia.” New England Journal of Medicine, 18 September 2024, doi: 10.1056/NEJMoa2312749.
Link: https://www.nejm.org/doi/full/10.1056/NEJMoa2312749
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