The use of anti-fibrinolytic agents in patients with HHT: a retrospective survey

The anti-fibrinolytic agent tranexamic acid (TXA) has been used to reduce bleeding in patients with hereditary hemorrhagic telangiectasia (HHT); however, there are limited data on its efficacy and safety. We conducted a retrospective study at the HHT center, using a structured questionnaire and data abstraction to determine the safety and effectiveness of TXA. Forty-two patients from the HHT database with a definite clinical or genetic diagnosis of HHT with epistaxis and/or gastrointestinal bleeding and receiving TXA were included, and their Epistaxis Severity Score (ESS), blood transfusions, and hospitalizations prior to and during TXA use were assessed. Among the 42 patients, 29 responded to the questionnaire (78.3 %). The ESS declined from a mean of 6.9 ± 2.7 to 4.3 ± 2.2 (p < 0.001), and there was a reduction in the percentage of patients receiving blood transfusion from 55.2 to 37.9 % (p = 0.095), hospitalizations from 58.6 to 37.9 % (p = 0.08), and medical care for epistaxis (i.e., clinic and/or emergency room visit) from 66.7 to 25.9 %. There was no difference in thrombosis (6.8 % before and during TXA use). TXA appears effective in the treatment of bleeding in patients with HHT, based on retrospective data. Prospective studies are warranted to confirm these preliminary results.