The effect of N-acetylcysteine on epistaxis and quality of life in patients with HHT: a pilot study

Rhinology. 2009 Mar;47(1):85-8.


Background: Free O2- radicals may cause precapillary sphincter abnormalities, resulting in epistaxis in hemizygous knockout mice for Endoglin. The objective of this study was to test if antioxidants, like N-acetylcysteine (NAC), are have a role in the treatment of epistaxis in hereditary hemorrhagic telangiectasia (HHT).

Methods: Forty-three patients participated in this study taking NAC 600 mg t.i.d for 12 weeks. Patients registered frequency, severity and duration of epistaxis and private and work-related quality of life (QOL), using a diary for two 6 weeks periods. The first period was prior to starting treatment and the second started after 6 weeks using NAC.

Results: There was a decrease infrequency (p < 0.01) and severity (p < 0.01) of epistaxis during the day. The improvement was most remarkable in male patients and patients with an ENDOGLIN mutation. In women and patients with an ALK-1 mutation, only a trend for improvement was found. Nocturnal epistaxis did not improve. The effect of epistaxis on the ability to work (p = 0.02) was reduced.

Conclusion: This pilot study was conducted to investigate whether animal experiments can be translated to humans with HHT regarding epistaxis. The positive results with NAC are promising and justify a randomised clinical trial.

Publication types

  • Clinical Trial

MeSH terms

  • Acetylcysteine / therapeutic use*
  • Epistaxis / etiology*
  • Epistaxis / prevention & control*
  • Female
  • Free Radical Scavengers / therapeutic use*
  • Humans
  • Male
  • Middle Aged
  • Pilot Projects
  • Quality of Life*
  • Severity of Illness Index
  • Sex Factors
  • Telangiectasia, Hereditary Hemorrhagic / complications*
  • Telangiectasia, Hereditary Hemorrhagic / drug therapy
  • Treatment Outcome


  • Free Radical Scavengers
  • Acetylcysteine