Topical Propranolol Improves Epistaxis Control in Hereditary Hemorrhagic Telangiectasia (HHT): A Randomized Double-Blind Placebo-Controlled Trial
Epistaxis is a common debilitating manifestation in hereditary hemorrhagic telangiectasia (HHT), due to mucocutaneous telangiectases. The epistaxis can be difficult to control despite available treatments. Dysregulated angiogenesis has been shown to be associated with telangiectases formation. Topic...
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MDPI AG
2020-09-01
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author | Meir Mei-Zahav Yulia Gendler Elchanan Bruckheimer Dario Prais Einat Birk Muhamad Watad Neta Goldschmidt Ethan Soudry |
author_facet | Meir Mei-Zahav Yulia Gendler Elchanan Bruckheimer Dario Prais Einat Birk Muhamad Watad Neta Goldschmidt Ethan Soudry |
author_sort | Meir Mei-Zahav |
collection | DOAJ |
description | Epistaxis is a common debilitating manifestation in hereditary hemorrhagic telangiectasia (HHT), due to mucocutaneous telangiectases. The epistaxis can be difficult to control despite available treatments. Dysregulated angiogenesis has been shown to be associated with telangiectases formation. Topical propranolol has demonstrated antiangiogenic properties. We performed a two-phase study, i.e., a double-blind placebo-controlled phase, followed by an open-label phase. The aim of the study was assessment of safety and efficacy of nasal propranolol gel in HHT-related epistaxis. Twenty participants with moderate-severe HHT-related epistaxis were randomized to eight weeks of propranolol gel 1.5%, or placebo 0.5 cc, applied to each nostril twice daily; and continued propranolol for eight weeks in an open-label study. For the propranolol group, the epistaxis severity score (ESS) improved significantly (−2.03 ± 1.7 as compared with −0.35 ± 0.68 for the placebo group, <i>p</i> = 0.009); hemoglobin levels improved significantly (10.5 ± 2.6 to 11.4 ± 2.02 g/dL, <i>p</i> = 0.009); and intravenous iron and blood transfusion requirement decreased. The change in nasal endoscopy findings was not significant. During the open-label period, the ESS score improved significantly in the former placebo group (−1.99 ± 1.41, <i>p</i> = 0.005). The most common adverse event was nasal mucosa burning sensation. No cardiovascular events were reported. Our results suggest that topical propranolol gel is safe and effective in HHT-related epistaxis. |
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spelling | doaj.art-8ae4edf9a68f4ece9d2a6d61134ce7f12023-11-20T15:21:07ZengMDPI AGJournal of Clinical Medicine2077-03832020-09-01910313010.3390/jcm9103130Topical Propranolol Improves Epistaxis Control in Hereditary Hemorrhagic Telangiectasia (HHT): A Randomized Double-Blind Placebo-Controlled TrialMeir Mei-Zahav0Yulia Gendler1Elchanan Bruckheimer2Dario Prais3Einat Birk4Muhamad Watad5Neta Goldschmidt6Ethan Soudry7Pulmonary Institute, Schneider Children’s Medical Center of Israel, Petah Tikva 49202, IsraelPulmonary Institute, Schneider Children’s Medical Center of Israel, Petah Tikva 49202, IsraelSackler Faculty of Medicine, Tel Aviv University, Tel Aviv 69978, IsraelPulmonary Institute, Schneider Children’s Medical Center of Israel, Petah Tikva 49202, IsraelSackler Faculty of Medicine, Tel Aviv University, Tel Aviv 69978, IsraelSackler Faculty of Medicine, Tel Aviv University, Tel Aviv 69978, IsraelDepartment of Hematology, Hadassah-Hebrew University Medical Center, Jerusalem 91120, IsraelSackler Faculty of Medicine, Tel Aviv University, Tel Aviv 69978, IsraelEpistaxis is a common debilitating manifestation in hereditary hemorrhagic telangiectasia (HHT), due to mucocutaneous telangiectases. The epistaxis can be difficult to control despite available treatments. Dysregulated angiogenesis has been shown to be associated with telangiectases formation. Topical propranolol has demonstrated antiangiogenic properties. We performed a two-phase study, i.e., a double-blind placebo-controlled phase, followed by an open-label phase. The aim of the study was assessment of safety and efficacy of nasal propranolol gel in HHT-related epistaxis. Twenty participants with moderate-severe HHT-related epistaxis were randomized to eight weeks of propranolol gel 1.5%, or placebo 0.5 cc, applied to each nostril twice daily; and continued propranolol for eight weeks in an open-label study. For the propranolol group, the epistaxis severity score (ESS) improved significantly (−2.03 ± 1.7 as compared with −0.35 ± 0.68 for the placebo group, <i>p</i> = 0.009); hemoglobin levels improved significantly (10.5 ± 2.6 to 11.4 ± 2.02 g/dL, <i>p</i> = 0.009); and intravenous iron and blood transfusion requirement decreased. The change in nasal endoscopy findings was not significant. During the open-label period, the ESS score improved significantly in the former placebo group (−1.99 ± 1.41, <i>p</i> = 0.005). The most common adverse event was nasal mucosa burning sensation. No cardiovascular events were reported. Our results suggest that topical propranolol gel is safe and effective in HHT-related epistaxis.https://www.mdpi.com/2077-0383/9/10/3130hereditary hemorrhagic telangiectasiaepistaxispropranolol gelepistaxis severity scorenasal endoscopyantiangiogenic properties |
spellingShingle | Meir Mei-Zahav Yulia Gendler Elchanan Bruckheimer Dario Prais Einat Birk Muhamad Watad Neta Goldschmidt Ethan Soudry Topical Propranolol Improves Epistaxis Control in Hereditary Hemorrhagic Telangiectasia (HHT): A Randomized Double-Blind Placebo-Controlled Trial Journal of Clinical Medicine hereditary hemorrhagic telangiectasia epistaxis propranolol gel epistaxis severity score nasal endoscopy antiangiogenic properties |
title | Topical Propranolol Improves Epistaxis Control in Hereditary Hemorrhagic Telangiectasia (HHT): A Randomized Double-Blind Placebo-Controlled Trial |
title_full | Topical Propranolol Improves Epistaxis Control in Hereditary Hemorrhagic Telangiectasia (HHT): A Randomized Double-Blind Placebo-Controlled Trial |
title_fullStr | Topical Propranolol Improves Epistaxis Control in Hereditary Hemorrhagic Telangiectasia (HHT): A Randomized Double-Blind Placebo-Controlled Trial |
title_full_unstemmed | Topical Propranolol Improves Epistaxis Control in Hereditary Hemorrhagic Telangiectasia (HHT): A Randomized Double-Blind Placebo-Controlled Trial |
title_short | Topical Propranolol Improves Epistaxis Control in Hereditary Hemorrhagic Telangiectasia (HHT): A Randomized Double-Blind Placebo-Controlled Trial |
title_sort | topical propranolol improves epistaxis control in hereditary hemorrhagic telangiectasia hht a randomized double blind placebo controlled trial |
topic | hereditary hemorrhagic telangiectasia epistaxis propranolol gel epistaxis severity score nasal endoscopy antiangiogenic properties |
url | https://www.mdpi.com/2077-0383/9/10/3130 |
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