ATERO : A Randomised Study With Tranexamic Acid in Epistaxis of Rendu Osler Syndrome

• Status: Completed
• Trial end date: 2010-05-01
• Target enrollment: 0
• Participant gender: All

Summary

Scientific Objectives Primary objective : Demonstrate that oral intake of tranexamic acid significantly reduces the risk of epistaxis occurrence, estimated by the average monthly duration of episodes of epistaxis.

Secondary objectives :

• Document the benefit of tranexamic acid on the amount of haemoglobine and quality of life of patients.

• Identify scalable and genetic factors of response to the treatment by tranexamic acid.

• Describe compliance and tolerance of the treatment. Method Experimental Design We suggest the realisation of a randomised comparative clinical trial versus placebo, with a crossover of random alternated periods of three months over a total of six months.

Study Population 213 affected patients, displaying sufficiently invalidating epistaxis to require a basal treatment, will be recruited and followed every 3 months. Recruitment will lean on teams who are part of the national French network in close collaboration with the reference centre for the Rendu-Osler disorder, appointed to Lyon on the 19th of November 2004. Follow up of the study will be carried out by the Clinical Investigational Centre of the related towns.

Outcome measures The main criterion of efficacy is the average duration of epistaxis, the secondary criterion of efficacy is the average number of epistaxis measured per month. Tolerance will be analysed according to the occurrence of venal or arterial thrombosis and allergic accidents. Venal thrombosis will systematically be sought by an inferior limbs echodoppler. Response markers will be sought through modelisation incorporating environmental, phenotypic and genetic factors.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Hospices Civils de Lyon
• Treatments: Tranexamic Acid

Criteria

Inclusion Criteria:

• Repeated, spontaneous epistaxis in a known Rendu Osler patient who wants global caring, these must be daily epistaxis (> 28 a month) or a total amount of 60 minutes of bleeding a month (50 % Rendu-Osler patients bleed before 20 and 99 % at 50 when penetrance is complete).

• Good completion of the grid of control by recording the number and duration of epistaxis over a period of three month.

Exclusion Criteria:

• Rendu Osler syndrome with specific phenotype : absence of epistaxis or occasional epistaxis, no need for particular care.

• Maintained expectations of an ENT treatment of epistaxis in a delay of three months.

• Incapacity of detailing the number of epistaxis, especially, at least, within three months before entering the study.

• No informed consent obtained after informing the patient on his participation to the study.

• Expected lack of observance because of an incapacity to compel to the daily treatment.

• Formal indication to Tranexamic Acid (angioneurotic oedema as a disease modifying treatment of 3 to 4 g/ day).

• Contra-indications to Tranexamic acid: history of convulsion, arterial or veinous thrombosis, , positivity of veinous echo doppler of the inferior limbs; Serum creatinine > 250 µmol/l.

N.B. Cases of bad observance are not frequent, patients being highly concerned with a treatment to reduce their discomfort and anaemia. They are more likely ready to move to a centre insuring this handling. Nevertheless, lack of observance will be detected over a period of 15 days of placebo in simple blind, before randomization.