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


CONCLUSIONS
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.
de Gussem
E M
EM
Department of Pulmonology, St Antonius Hospital, Nieuwegein, The Netherlands.
Snijder
R J
RJ
Disch
F J
FJ
Zanen
P
P
Westermann
C J J
CJ
Mager
J J
JJ
D000111:Acetylcysteine / Q000627:therapeutic use*; D004844:Epistaxis / Q000209:etiology* / Q000517:prevention & control*; D005260:Female; D016166:Free Radical Scavengers / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D008875:Middle Aged; D010865:Pilot Projects; D011788:Quality of Life; D012720:Severity of Illness Index; D012737:Sex Factors; D013683:Telangiectasia, Hereditary Hemorrhagic / Q000150:complications* / Q000188:drug therapy; D016896:Treatment Outcome
D016166:Free Radical Scavengers; D000111:Acetylcysteine