David Green, MD, PhD

This fibrinolysis inhibitor was modestly effective and well tolerated.

Persons with hereditary hemorrhagic telangiectasia (HHT; Rendu-Osler-Weber disease) have recurrent episodes of bleeding from telangiectasias in the nose and internal organs. Tranexamic acid is an inhibitor of fibrinolysis that has been reported to decrease bleeding in this disorder.

To examine the efficacy and safety of tranexamic acid in HHT, European investigators conducted a multicenter, randomized, placebo-controlled, double-blind, crossover trial involving 118 patients who received tranexamic acid (1.5 g twice daily) followed by placebo or the opposite sequence; each treatment period was 3 months.

The mean duration of nose-bleeding each month (the primary endpoint) was shorter during treatment with tranexamic acid versus placebo (106 vs. 122 minutes; P=0.005). Although the median number of episodes of nose-bleeding was similar during both treatments (23 and 22), it was decreased by 5.5% with tranexamic acid if analyzed by the paired-data method (P=0.0005). However, there was no change in hemoglobin level and no improvement in quality of life with either treatment. The main adverse effects of tranexamic acid versus placebo were vertigo (17.5% vs. 3.3%) and diarrhea (18.0% vs. 5.3%).

Citation(s):

Gaillard S et al. Tranexamic acid for epistaxis in hereditary hemorrhagic telangiectasia patients: A European cross-over controlled trial in a rare disease. J Thromb Haemost 2014; 12:1494.