Pazopanib Effects on Bleeding in Hereditary Hemorrhagic Telangiectasia

Conditions:   Hereditary Hemorrhagic Telangiectasia;   Epistaxis Nosebleed;   Anemia Interventions:   Drug: Pazopanib;   Drug: Placebo oral capsule Sponsor:   Cure HHT Not yet recruiting
Source: - Category: Research Source Type: clinical trials

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This study was carried out in accordance with approval of the Melbourne Health and Walter and Eliza Hall Institute of Medical Research's Human Research Ethics Committee (approval number: 2013.081). All subjects gave written informed consent for participation and publication. Results and Discussion TGF-β signaling in NK cells is associated with: phosphorylation in SMAD2 and 3, inhibition of IL-15-induced metabolism/proliferation, simultaneous downregulation of CD44, CD49e, and Eomes, and upregulation of CD16 and CD49a expression (7, 10). SMAD family member 4 (SMAD4) belongs to the SMAD family of transcription...
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
Pulmonary arteriovenous malformations (PAVMs) are rare and commonly caused by an autosomal dominant disorder known as hereditary hemorrhagic telangiectasia (HHT). HHT is an often-missed vascular disorder causing symptoms of epistaxis, mucosal telangiectasia, AVMs, hemoptysis, dyspnea, and iron deficiency anemia. Large AVMs in the lungs, liver, and brain can be life-threatening. Early diagnosis and intervention are imperative to prevent serious complications of massive hemoptysis, stroke, cerebral abscess, and gastrointestinal bleeding.
Source: The Journal for Nurse Practitioners - Category: Nursing Authors: Tags: Featured Article Source Type: research
Introduction: Hereditary hemorrhagic telangiectasia (HHT) is a rare hereditary multisystem vascular disorder causing visceral arteriovenous malformations (AVMs) and mucocutaneous bleeding. Chronic gastrointestinal bleeding and epistaxis frequently produce profound iron deficiency anemia refractory to conventional treatment. Patients with HHT have elevations in vascular endothelial growth factor (VEGF), so the anti-VEGF agent bevacizumab, a recombinant, humanized monoclonal IgG1 antibody that binds to and neutralizes circulating VEGF, is a promising systemic HHT therapy. Currently, data pertaining to the efficacy of bevaciz...
Source: Blood - Category: Hematology Authors: Tags: 102. Regulation of Iron Metabolism: Translating Iron Biology to the Clinic Source Type: research
In conclusion, we observed an improvement in Hgb and/or epistaxis in all treated patients. This occurred at a dose much lower than typically used for oncologic indications, with no serious AE. Further studies of pazopanib efficacy are warranted.
Source: Angiogenesis - Category: Molecular Biology Source Type: research
This study is designed to analyze the clinical features and treatment options for patients with HHT. Hospitalized patients with a definitive diagnosis of HHT from November 1973 to July 2016 in Peking Union Medical College Hospital were identified after reviewing medical records and electronic databases. Further follow-up data of these patients were collected from outpatient clinical visits and/or telephone interviews. We identified a total of 20 patients, 7 males and 13 females. The mean age was 42.4 ± 20.3 years. Epistaxis (18/20) was the most common presentation, followed by telangiectases of the oral buccal...
Source: Medicine - Category: Internal Medicine Tags: Research Article: Observational Study Source Type: research
Hereditary hemorrhagic telangiectasia (HHT) is an autosomal dominant disorder with a worldwide prevalence rate of approximately 1 case per 5000 persons that results in vascular malformations throughout the body. At least 90% of patients are affected by nosebleeds and up to 81% have gastrointestinal telangiectasias.1 Both of these can result in severe bleeding and anemia that are refractory to conventional therapy (eg, iron infusions and endoscopic cautery of gastrointestinal and nasal telangiectasias) in perhaps as many as 10% to 15% of patients.
Source: Mayo Clinic Proceedings - Category: Internal Medicine Authors: Tags: Editorial Source Type: research
Hereditary hemorrhagic telangiectasia (HHT) is an inherited disease characterized by mucocutaneous telangiectatic lesions, arteriovenous malformations and recurrent spontaneous epistaxis. A 61-year-old white male patient sought dental service because of a recurrent gingival bleeding. He had previously been diagnosed with HHT. His medical history included pulmonary hypertension, brain abscess, arterial hypertension, iron deficiency anemia, recurrent episodes of epistaxis and oral bleeding. Physical examination revealed numerous telangiectatic lesions on the face, tongue, buccal mucosa, palate, gums and a vascular malformati...
Source: Oral Surgery, Oral Medicine, Oral Pathology, Oral Radiology, and Endodontics - Category: ENT & OMF Authors: Tags: CPP - Clinical Poster Presentation Source Type: research
CONCLUSIONS: Sorafenib and GW771806 significantly improved, yet erlotinib worsened anemia and GI-bleeding in the Alk1-iKO model. However, none of these TKIs appeared to be effective for inhibiting the development of wound-induced skin AVMs. Taken together, these results suggest that oral delivery of anti-angiogenic TKIs are selectively more effective on GI bleeding than mucocutaneous AVMs, and it may provide an experimental basis for selective therapeutic options depending on the symptoms of HHT. This article is protected by copyright. All rights reserved. PMID: 28339142 [PubMed - as supplied by publisher]
Source: Thrombosis and Haemostasis - Category: Hematology Authors: Tags: J Thromb Haemost Source Type: research
CONCLUSION: Intermittent low-dose therapy with bevacizumab represents an effective and cost-efficient treatment option for transfusion-dependent patients with hereditary hemorrhagic telangiectasia. PMID: 27878613 [PubMed - as supplied by publisher]
Source: Wiener Klinische Wochenschrift - Category: Journals (General) Authors: Tags: Wien Klin Wochenschr Source Type: research
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