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...

Full description

Bibliographic Details
Main Authors: Meir Mei-Zahav, Yulia Gendler, Elchanan Bruckheimer, Dario Prais, Einat Birk, Muhamad Watad, Neta Goldschmidt, Ethan Soudry
Format: Article
Language:English
Published: MDPI AG 2020-09-01
Series:Journal of Clinical Medicine
Subjects:
Online Access:https://www.mdpi.com/2077-0383/9/10/3130
id doaj-8ae4edf9a68f4ece9d2a6d61134ce7f1
record_format Article
spelling doaj-8ae4edf9a68f4ece9d2a6d61134ce7f12020-11-25T03:00:06ZengMDPI AGJournal of Clinical Medicine2077-03832020-09-0193130313010.3390/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
collection DOAJ
language English
format Article
sources DOAJ
author Meir Mei-Zahav
Yulia Gendler
Elchanan Bruckheimer
Dario Prais
Einat Birk
Muhamad Watad
Neta Goldschmidt
Ethan Soudry
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
author_facet Meir Mei-Zahav
Yulia Gendler
Elchanan Bruckheimer
Dario Prais
Einat Birk
Muhamad Watad
Neta Goldschmidt
Ethan Soudry
author_sort Meir Mei-Zahav
title 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_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_sort topical propranolol improves epistaxis control in hereditary hemorrhagic telangiectasia (hht): a randomized double-blind placebo-controlled trial
publisher MDPI AG
series Journal of Clinical Medicine
issn 2077-0383
publishDate 2020-09-01
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.
topic hereditary hemorrhagic telangiectasia
epistaxis
propranolol gel
epistaxis severity score
nasal endoscopy
antiangiogenic properties
url https://www.mdpi.com/2077-0383/9/10/3130
work_keys_str_mv AT meirmeizahav topicalpropranololimprovesepistaxiscontrolinhereditaryhemorrhagictelangiectasiahhtarandomizeddoubleblindplacebocontrolledtrial
AT yuliagendler topicalpropranololimprovesepistaxiscontrolinhereditaryhemorrhagictelangiectasiahhtarandomizeddoubleblindplacebocontrolledtrial
AT elchananbruckheimer topicalpropranololimprovesepistaxiscontrolinhereditaryhemorrhagictelangiectasiahhtarandomizeddoubleblindplacebocontrolledtrial
AT darioprais topicalpropranololimprovesepistaxiscontrolinhereditaryhemorrhagictelangiectasiahhtarandomizeddoubleblindplacebocontrolledtrial
AT einatbirk topicalpropranololimprovesepistaxiscontrolinhereditaryhemorrhagictelangiectasiahhtarandomizeddoubleblindplacebocontrolledtrial
AT muhamadwatad topicalpropranololimprovesepistaxiscontrolinhereditaryhemorrhagictelangiectasiahhtarandomizeddoubleblindplacebocontrolledtrial
AT netagoldschmidt topicalpropranololimprovesepistaxiscontrolinhereditaryhemorrhagictelangiectasiahhtarandomizeddoubleblindplacebocontrolledtrial
AT ethansoudry topicalpropranololimprovesepistaxiscontrolinhereditaryhemorrhagictelangiectasiahhtarandomizeddoubleblindplacebocontrolledtrial
_version_ 1724699326293213184