Urban W Geisthoff1, Ulrich T Seyfert2, Marcus Kübler3, Birgitt Bieg4, Peter K Plinkert5, Jochem König6. 1. Department of Otorhinolaryngology, Essen University Hospital, Germany; Morbus Osler-Selbsthilfe e.V. German HHT self-help group, Niederkrüchten, Germany; Formerly of: Department of Otorhinolaryngology, Hospitals of the University of the Saarland, Homburg/Saar, Germany. Electronic address: http://geisthoff.de. 2. Medical Practice for Haemostaseology and Transfusion Medicine, Saarbrücken, Germany; Formerly of: Department of Haemostaseology and Transfusion Medicine, Hospitals of the University of the Saarland, Homburg/Saar, Germany. 3. Formerly of: Department of Otorhinolaryngology, Hospitals of the University of the Saarland, Homburg/Saar, Germany; B.A.D. Gesundheitsvorsorge und Sicherheitstechnik GmbH, Team Prevent, Munich, Germany; Medical Practice Dr. Rolf Kübler, Stuttgart, Germany. 4. Formerly of: Department of Haemostaseology and Transfusion Medicine, Hospitals of the University of the Saarland, Homburg/Saar, Germany; Department of Anaesthesiology, Klinikum Braunschweig, Braunschweig, Germany. 5. Formerly of: Department of Otorhinolaryngology, Hospitals of the University of the Saarland, Homburg/Saar, Germany; Department of Otorhinolaryngology, Ruprecht-Karls-University, Heidelberg, Germany. 6. Institute of Medical Biostatistics, Epidemiology and Informatics, Division Medical Biometry, University Medical Center, Johannes Gutenberg University, Mainz, Germany; Formerly of: Institute of Medical Biostatistics, Epidemiology and Informatics, University of the Saarland, Homburg/Saar, Germany.
Abstract
INTRODUCTION: Epistaxis is the most frequent manifestation in hereditary hemorrhagic telangiectasia, in which no optimal treatment exists. It can lead to severe anemia and reduced quality of life. Positive effects of tranexamic acid, an antifibrinolytic drug, have been reported on epistaxis related to this disorder. We sought to evaluate the efficacy of treating nosebleeds in hereditary hemorrhagic telangiectasia with tranexamic acid. MATERIALS AND METHODS: In a randomized, double-blind, placebo controlled, cross-over phase IIIB study, 1 gram of tranexamic acid or placebo was given orally 3 times daily for 3 months for a total of 6 months. RESULTS:22 patients were included in the intention-to-treat analysis. Hemoglobin levels, the primary outcome measure, did not change significantly (p=0.33). The secondary outcome measure was epistaxis score and patients reported a statistically significant reduction in nosebleeds, equaling a clinically relevant 54% diminution (p=0.0031), as compared to the placebo period. No severe side effects were observed. CONCLUSION:Tranexamic acid reduces epistaxis in patients with hereditary hemorrhagic telangiectasia. (Clinical trial registration numbers: BfArM 141 CHC 9008-001 and ClinicalTrials.gov NCT01031992).
RCT Entities:
INTRODUCTION: Epistaxis is the most frequent manifestation in hereditary hemorrhagic telangiectasia, in which no optimal treatment exists. It can lead to severe anemia and reduced quality of life. Positive effects of tranexamic acid, an antifibrinolytic drug, have been reported on epistaxis related to this disorder. We sought to evaluate the efficacy of treating nosebleeds in hereditary hemorrhagic telangiectasia with tranexamic acid. MATERIALS AND METHODS: In a randomized, double-blind, placebo controlled, cross-over phase IIIB study, 1 gram of tranexamic acid or placebo was given orally 3 times daily for 3 months for a total of 6 months. RESULTS: 22 patients were included in the intention-to-treat analysis. Hemoglobin levels, the primary outcome measure, did not change significantly (p=0.33). The secondary outcome measure was epistaxis score and patients reported a statistically significant reduction in nosebleeds, equaling a clinically relevant 54% diminution (p=0.0031), as compared to the placebo period. No severe side effects were observed. CONCLUSION:Tranexamic acid reduces epistaxis in patients with hereditary hemorrhagic telangiectasia. (Clinical trial registration numbers: BfArM 141 CHC 9008-001 and ClinicalTrials.gov NCT01031992).