Drug Therapy
Drugs are approved in the U.S. by the Food and Drug Administration (FDA) for specific purposes to help guide medical practice.
Currently, there are no FDA approved drugs specifically for the treatment of HHT. However, there have been several promising studies and positive results for medications originally developed to treat other conditions.
Related Resources
Pazopanib: A Groundbreaking Trial for HHT (Recording)
Webinar – 2023Get your questions answered about our newest clinical trial! Join Cassi Friday, PhD and Jim Gossage, MD as they explain who is eligible, what Pazopanib is and how you can get involved!
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Research Roundtable: An Inside Look at Exciting Clinical Trials
Webinar – 2023Delve into the exciting clinical research projects underway in HHT! Cassi Friday, PhD will provide an overview of what goes into a clinical trial. Dr. Marie Faughnan (Brain AVM Study), Dr. Keith McCrae (PATH Study), Dr. James Gossage (Pazopanib Trial…
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Bedside to Bench: How the HHT Community Drives Research Initiatives (Recorded)
Webinar – 2021In March 2020, Cure HHT was 1 of 30 organizations selected to participate in the Chan Zuckerberg Initiative (also known as CZI) Rare is One Project. This ambitious project is lifting up the work that patient communities are doing to accelerate resear…
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Nuts and Bolts of A Clinical Trial – the PATH-HHT Study (Recorded)
Webinar – 2021Research is the key to the future health and quality of life for HHT patients. “Diseases can only be cured when scientists understand them. This understanding, which comes from basic research, leads to new drugs and treatments.” ~Dr. Christopher Hugh…
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International HHT Guidelines – Overview (Recorded)
Webinar – 2021Join Dr. Marie Faughnan, Director, Toronto HHT Centre of Excellence as she provides an overview of the newly updated International HHT Guidelines and how they are used to set the standard of care globally for the diagnosis and treatment of HHT. Dr. F…
MoreBevacizumab (Avastin) Use In HHT
Policy Statement – 2020Hereditary hemorrhagic telangiectasia (HHT, also known as OslerWeber-Rendu Syndrome) is a condition that is characterized by focal proliferation of blood vessels (vascular malformations) in multiple organs. Two of the most commonly affected organs in…
MoreBevacizumab for treating HHT patients with severe hepatic involvement or refractory anemia
Scientific Literature – 2020PLoS One. Authors: Carolina Vázquez, María Laura Gonzalez, Augusto Ferraris, Juan Carlos Bandi, and Marcelo Martín Serra. This article is available for public use and does not require purchase. Click to view the full article.
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The Science Behind the 2019 Scientific Conference (recorded)
Webinar – 2020Philippe Marambaud, PhD and Andy White, MD discuss important advances in HHT research which were presented at the 2019 HHT International Scientific Conference in Puerto Rico. Learn about advancements in drug therapies for bleeding, screening and trea…
MoreSystemic bevacizumab for high-output cardiac failure in HHT: an international survey of HHT centers
Scientific Literature – 2019Orphanet Journal of Rare Diseases. Authors: Hanny Al-Samkari, Hasan A. Albitar, Scott E. Olitsky, Marianne S. Clancy and Vivek N. Iyer. This article is available for public use and does not require purchase. Click to view the full article.
MoreSystemic bevacizumab for the treatment of chronic bleeding in HHT
Scientific Literature – 2018Journal of Internal Medicine. Authors: Al-Samkari, H., Kritharis, A., Rodriguez-Lopez, J.M., and Kuter, D.J. This article can be purchased directly from the publisher. Click to view the article description and purchase options.
MoreHHT: Diagnosis and Management from the Hematologist’s Perspective
Scientific Literature – 2018Haematologica. Authors: Athena Kritharis, Hanny Al-Samkari, and David J Kuter. This article is available for public use and does not require purchase. Click to view the full article.
MoreThe Current Role of Bevacizumab in the Treatment of Hereditary Hemorrhagic Telangiectasia Related Bleeding
Scientific Literature – 2018Mayo Clinic. Author: James R. Gossage, MD This article is available for public use and does not require purchase. Click to view the full article.
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HHT Scientific Conference Summaries (recorded)
Webinar – 2018Drs. Hughes, Faughnan and Andrejecsk provide a first hand synopsis of the HHT science presented in Dubrovnik, Croatia where over 200 voices were heard from 23 countries. They reviewed the 12th HHT International Conference Executive Summary of…
MoreExecutive Summary – 12th HHT International Scientific Conference (2017)
Scientific Literature – 2017Angiogenesis. Authors: Jillian W. Andrejecsk, Anna E. Hosman, Luisa M. Botella, Claire L. Shovlin, Helen M. Arthur, Sophie Dupuis-Girod, Elisabetta Buscarini, Christopher C. W. Hughes, Franck Lebrin, Christine L. Mummery, Marco C. Post, and Johannes…
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Hazardous Natural Health Products in HHT
Article – 2017HHT patients can bleed easily, which is why products that may thin your blood or reduce your ability to clot can be very dangerous. This handout was developed by Daniel Cortes, Pharmacist at the St. Michael’s Hospital HHT Center in Toronto, Canada, t…
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