ArticleAngiogenesis2022
Pazopanib for severe bleeding and transfusion-dependent anemia in hereditary hemorrhagic telangiectasia.
Article in Angiogenesis, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03850964 (A Phase II/III Randomized, Placebo Controlled, Double Blind Study to Evaluate the Effects of up to 24 Weeks of Low Dose Pazopanib on Hereditary Hemorrhagic Telangiectasia Related Epistaxis and Anemia), which is not on this map. Cited by 31 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase II/III Randomized, Placebo Controlled, Double Blind Study to Evaluate the Effects of up to 24 Weeks of Low Dose Pazopanib on Hereditary Hemorrhagic Telangiectasia Related Epistaxis and Anemia
Who cites it
31 citing papers in PubMed, 47 citations in OpenAlex.
- Characteristics associated with clinical response to pomalidomide in hereditary hemorrhagic telangiectasia.Blood advances · 2026Trial
- Effect of oral nintedanib vs placebo on epistaxis in hereditary hemorrhagic telangiectasia: the EPICURE multicenter randomized double-blind trial.Angiogenesis · 2024Trial
- Randomized, double-blind, placebo-controlled, crossover trial of oral doxycycline for epistaxis in hereditary hemorrhagic telangiectasia.Orphanet journal of rare diseases · 2022Trial
- Systemic Bevacizumab for Severe Bleeding From Acquired Gastrointestinal Vascular Malformations.American journal of hematology · 2026Observational
- Patients with hereditary hemorrhagic telangiectasia have significantly reduced overall survival-And likely by a greater magnitude than we realize.Journal of internal medicine · 2026Article
- A microphysiological system HHT-on-a-chip platform recapitulates patient vascular lesions.Nature communications · 2026Article
- Article
- Pomalidomide for hereditary hemorrhagic telangiectasia: after trial longitudinal assessment study (PATH-HHT ATLAS).Blood advances · 2026Observational
- Long-term outcomes of blue light laser therapy for epistaxis in patients with hereditary hemorrhagic telangiectasia within a multimodal treatment concept.European archives of oto-rhino-laryngology : official journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery · 2026Article
- Propranolol Reduces Epistaxis in Hereditary Hemorrhagic Telangiectasia: A Large Retrospective Study.Journal of clinical medicine · 2026Article
- Validation and clinical application of the hereditary hemorrhagic telangiectasia-specific quality of life scale.Journal of thrombosis and haemostasis : JTH · 2026Article
- Epistaxis Prevention, Treatment, and Future Perspectives for Hereditary Hemorrhagic Telangiectasia.Journal of clinical medicine · 2025Review
- Characterizing the Healthcare Utilization and Costs of Hereditary Hemorrhagic Telangiectasia.American journal of hematology · 2025Article
- Standardization of Terminology, Definitions, and Outcome Criteria for Bleeding in Hereditary Hemorrhagic Telangiectasia: International Consensus Report.American journal of hematology · 2025Review
- Epistaxis due to hereditary hemorrhagic telangiectasia: A case report and literature review.The Journal of international medical research · 2025Review
- Hemoptysis caused by hereditary hemorrhagic telangiectasia: A case report and literature review.Zhong nan da xue xue bao. Yi xue ban = Journal of Central South University. Medical sciences · 2025Review
- Hereditary haemorrhagic telangiectasia.Nature reviews. Disease primers · 2025Review
- Potential and emerging therapeutics for HHT.Hematology. American Society of Hematology. Education Program · 2024Review
- Pulmonary vascular manifestations of hereditary haemorrhagic telangiectasia.Pulmonary circulation · 2024Review
- Hereditary Hemorrhagic Telangiectasia (Osler's Disease): Systemic, Interdisciplinary, Relatively Common—and Often Missed.Deutsches Arzteblatt international · 2024Review
Corrections and comments
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Authors and funding
9 authors at 4 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Hereditary hemorrhagic telangiectasia (HHT) is a rare angiogenic disorder causing chronic gastrointestinal bleeding, epistaxis, and severe anemia. Pazopanib is an oral multi-kinase angiogenesis inhibitor with promise to treat bleeding in HHT. We analyzed outcomes of HHT patients with the most severe bleeding causing RBC transfusion dependence treated on a predefined institutional pazopanib treatment pathway (with data collected retrospectively). The primary endpoint was achievement of transfusion independence. Secondary endpoints included hemoglobin, epistaxis severity score, RBC transfusion and iron infusion requirements, number of local hemostatic procedures, ferritin and transferrin saturation, compared using paired and repeated measures mean tests. Thirteen transfusion-dependent HHT patients received pazopanib [median (range) dose 150 (25-300) mg daily)] for a median of 22 months. All patients achieved transfusion independence. Compared with pretreatment, pazopanib increased mean hemoglobin by 4.8 (95% CI, 3.6-5.9) g/dL (7.8 vs. 12.7 g/dL, P < 0.0001) and decreased mean epistaxis severity score by 4.77 (3.11-6.44) points (7.20 vs. 2.43 points, P < 0.0001) after 12 months of treatment. Compared with 3 months of pretreatment, RBC transfusions decreased by 93% (median of 16.0 vs. 0.0 units, P < 0.0001) and elemental iron infusion decreased by 92% (median of 4500 vs. 0 mg, P = 0.005) during the first 3 months of treatment; improvements were maintained over time. Pazopanib was well-tolerated: hypertension, lymphocytopenia, and fatigue were the most common TEAEs. In conclusion, pazopanib was safe and effective to manage severe bleeding in HHT, liberating all patients from transfusion dependence and normalizing hematologic parameters at doses lower than used to treat malignancies. These findings require confirmation in a randomized trial.
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