PB0579 - Safety of continuous long term antifibrinolytic therapy in patients with Hereditary Hemorrhagic Telangiectasia

Background: Hereditary Hemorrhagic Telangiectasia (HHT) is an inherited bleeding disorder characterized by formation of mucocutaneous telangiectasias and visceral organ arteriovenous malformations. Affected individuals develop recurrent spontaneous epistaxis and gastrointestinal bleeding, which can be life threatening. Iron deficiency anemia develops in 50% of individuals, requiring frequent iron infusions and occasional blood transfusions. Antifibrinolytics are commonly used to reduce the frequency and severity of bleeding in HHT. However, safety of its long-term use has not been evaluated.

Aims: Determine the long-term safety of continuous antifibrinolytic therapy in patients with HHT.

Methods: This is a single institution retrospective study approved by the Institutional Review Board. Electronic medical records were reviewed to obtain patient demographics, antifibrinolytics use, adverse effects, and efficacy (epistaxis severity score, hemoglobin). Only patients on continuous antifibrinolytic therapy were included.

Results: Sixty seven patients were included in the study. Most were female, white, with median age of 66 years, and on tranexamic acid. Seventy five % were on antifibrinolytics for more than a year, with 28% longer than 5 years. Antifibrinolytics were discontinued in 40% of patients, 62% of which was within the first year. Discontinuation was most commonly due to efficacy (12%), lack of efficacy (9%), or treatment-related adverse effects (TAEs; 7%). The most common TAEs were headache, gastrointestinal intolerance and visual disturbances. Incidence of venous thromboembolism was 4%, with all of them occurring within 5 years of therapy, two of them after recent hospital admission (Table 2). Data from an additional 99 subjects on continuous antifibrinolytics are under evaluation.

Conclusion(s): This is the largest report on continuous antifibrinolytic therapy to date. Majority of patients remain on therapy. Antifibrinolytics were well tolerated with discontinuation due to TAEs occurring in only 7%. Incidence of VTE is low. Long term use of antifibrinolytics in patients with HHT is safe and effective.