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A randomised, double-blind, placebo-controlled trial of oral propranolol for hereditary haemorrhagic telangiectasia

Liaw, Joshua ; Cervin, Anders LU ; Grigg, Cameron ; Liu, Zhen ; Bates, James ; Brahmabhatt, Pranter ; Girling, Kathryn ; Banks, Alecia ; McCormack, Louise and Walker, Abigail , et al. (2025) In Australian Journal of Otolaryngology 8.
Abstract

Background: Early evidence suggests beta-blockers may have a disease-modifying effect in hereditary haemorrhagic telangiectasia (HHT). We aimed to assess the efficacy of oral propranolol is reducing severity of HHT-related epistaxis. Methods: A randomised, double blind, placebo controlled clinical trial was conducted in adults with HHT experiencing epistaxis. Subjects were randomly assigned to receive 40 mg twice-daily oral propranolol or placebo for 6 months with an additional month of observation post treatment cessation. The primary outcome was the validated Epistaxis Severity Score (ESS), with secondary outcomes measures including quality of life, endoscopic scoring, and biochemistry markers. Results: Twenty-three patients were... (More)

Background: Early evidence suggests beta-blockers may have a disease-modifying effect in hereditary haemorrhagic telangiectasia (HHT). We aimed to assess the efficacy of oral propranolol is reducing severity of HHT-related epistaxis. Methods: A randomised, double blind, placebo controlled clinical trial was conducted in adults with HHT experiencing epistaxis. Subjects were randomly assigned to receive 40 mg twice-daily oral propranolol or placebo for 6 months with an additional month of observation post treatment cessation. The primary outcome was the validated Epistaxis Severity Score (ESS), with secondary outcomes measures including quality of life, endoscopic scoring, and biochemistry markers. Results: Twenty-three patients were included, with 15 patients completing the treatment period. At 3 months, patients in the propranolol group showed a statistically significant reduction in ESS compared to baseline [mean change: −1.68, 95% confidence interval (CI): −3.25 to −0.12, P=0.038]. This trend continued at 6 months (mean change −1.44, 95% CI: −2.98 to 0.10, P=0.064) but was not statistically significant. The net change in ESS between treatments did not reach statistical significance at 6 months (P=0.254). Patients with higher baseline ESS scores demonstrated greater improvement, as well as improved emotional and social functioning domains. Hemoglobin (Hb) levels increased significantly at 3 months (mean increase: 7.7 g/L, 95% CI: 1.57 to 13.93, P=0.018) before returning to baseline at 6 months in the treatment group. No significant differences were observed in secondary outcomes, including endoscopic scores and Short Form-36 (SF-36) quality-of-life domains. Hypotension and associated dizziness occurred in three patients on propranolol, though no physical injury or major adverse event was reported. Conclusions: Oral propranolol may provide a safe temporary reduction in epistaxis severity at 3 months, but further research is needed to assess long-term benefits and optimal dosing strategies. Study limitations include a small sample size and possible selection bias. Larger, multi-center trials are required for confirmation. Trial Registration: Australian New Zealand Clinical Trials Registry; ANZCTR; registration ID: ACTRN12619001020178.

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Contribution to journal
publication status
published
subject
keywords
adrenergic beta-antagonists, arteriovenous malformation, Epistaxis, Osler-Rendu-Weber disease
in
Australian Journal of Otolaryngology
volume
8
article number
26
publisher
AME Publishing Company
external identifiers
  • scopus:105020640049
ISSN
2616-2792
DOI
10.21037/ajo-24-64
language
English
LU publication?
yes
additional info
Publisher Copyright: © AME Publishing Company, Australian Society of Otolaryngology Head & Neck Surgery.
id
8eb2cafc-1409-4106-8ac3-be9a29a82036
date added to LUP
2025-12-19 12:59:34
date last changed
2025-12-19 12:59:51
@article{8eb2cafc-1409-4106-8ac3-be9a29a82036,
  abstract     = {{<p>Background: Early evidence suggests beta-blockers may have a disease-modifying effect in hereditary haemorrhagic telangiectasia (HHT). We aimed to assess the efficacy of oral propranolol is reducing severity of HHT-related epistaxis. Methods: A randomised, double blind, placebo controlled clinical trial was conducted in adults with HHT experiencing epistaxis. Subjects were randomly assigned to receive 40 mg twice-daily oral propranolol or placebo for 6 months with an additional month of observation post treatment cessation. The primary outcome was the validated Epistaxis Severity Score (ESS), with secondary outcomes measures including quality of life, endoscopic scoring, and biochemistry markers. Results: Twenty-three patients were included, with 15 patients completing the treatment period. At 3 months, patients in the propranolol group showed a statistically significant reduction in ESS compared to baseline [mean change: −1.68, 95% confidence interval (CI): −3.25 to −0.12, P=0.038]. This trend continued at 6 months (mean change −1.44, 95% CI: −2.98 to 0.10, P=0.064) but was not statistically significant. The net change in ESS between treatments did not reach statistical significance at 6 months (P=0.254). Patients with higher baseline ESS scores demonstrated greater improvement, as well as improved emotional and social functioning domains. Hemoglobin (Hb) levels increased significantly at 3 months (mean increase: 7.7 g/L, 95% CI: 1.57 to 13.93, P=0.018) before returning to baseline at 6 months in the treatment group. No significant differences were observed in secondary outcomes, including endoscopic scores and Short Form-36 (SF-36) quality-of-life domains. Hypotension and associated dizziness occurred in three patients on propranolol, though no physical injury or major adverse event was reported. Conclusions: Oral propranolol may provide a safe temporary reduction in epistaxis severity at 3 months, but further research is needed to assess long-term benefits and optimal dosing strategies. Study limitations include a small sample size and possible selection bias. Larger, multi-center trials are required for confirmation. Trial Registration: Australian New Zealand Clinical Trials Registry; ANZCTR; registration ID: ACTRN12619001020178.</p>}},
  author       = {{Liaw, Joshua and Cervin, Anders and Grigg, Cameron and Liu, Zhen and Bates, James and Brahmabhatt, Pranter and Girling, Kathryn and Banks, Alecia and McCormack, Louise and Walker, Abigail and Earnshaw, James}},
  issn         = {{2616-2792}},
  keywords     = {{adrenergic beta-antagonists; arteriovenous malformation; Epistaxis; Osler-Rendu-Weber disease}},
  language     = {{eng}},
  month        = {{06}},
  publisher    = {{AME Publishing Company}},
  series       = {{Australian Journal of Otolaryngology}},
  title        = {{A randomised, double-blind, placebo-controlled trial of oral propranolol for hereditary haemorrhagic telangiectasia}},
  url          = {{http://dx.doi.org/10.21037/ajo-24-64}},
  doi          = {{10.21037/ajo-24-64}},
  volume       = {{8}},
  year         = {{2025}},
}