Pazopanib for Herditary Hemorrhaigic Telamgiectasia ( HHT) – pro
Hereditary Hemorrhagic Telangiectasia (HHT) has no currently established medical treatment. Surgical options are largely palliative, with variable and temporary results. and Avastin is often sued based on case reports. Pazopanib was recently reported from thirteen transfusion-dependent HHT patients who received pazopanib [median (range) dose 150 (25-300) mg daily)] for a median of 22 months. All patients achieved transfusion independence and the treatment was well-tolerated. However, the authors (Parambil et al) said that these findings require confirmation in a randomized trial. A Phase II/III clinical trial was launched in October 2023.
W Shovlin C, Ganesan V. Hereditary hemorrhagic telangiectasia (Osler-Weber-Rendu syndrome). In: UpToDate, Basow, DS (Ed), UpToDate, Waltham, MA, 2014. Literature review current through October 2014
Shovlin CL. Hereditary haemorrhagic telangiectasia: pathophysiology, diagnosis and treatment. Blood Rev 2010; 24:203.
https://www.bio.uci.edu/new-clinical-trial-for-pazopanib-offers-hope-for-hht-patients/, Accessed March 14, 2024
Parambil JG, Gossage JR, McCrae KR, Woodard TD, Menon KVN, Timmerman KL, Pederson DP, Sprecher DL, Al-Samkari H. Pazopanib for severe bleeding and transfusion-dependent anemia in hereditary hemorrhagic telangiectasia. Angiogenesis. 2022 Feb;25(1):87-97.