Georges Brohee Prize. Oestrogen-progesterone, a new therapy of bleeding gastrointestinal vascular malformations.

Gastrointestinal vascular malformations can cause haemorrhage requiring multiple transfusions. Surgical or endoscopic therapy are ineffective when the vascular malformations are spread diffusely over the gastrointestinal tract, when lesions escape identification or are not accessible for treatment. Several reports suggest that oestrogen-progesterone therapy is effective in the treatment of epistaxis in hereditary haemorrhagic telangiectasia. The aim of our studies is to test the hypothesis that oestrogen-progesterone is an effective treatment for bleeding gastrointestinal vascular malformations in patients with a very high transfusion need and in whom surgical or endoscopic treatment failed or could not be performed. The mean transfusion requirement prior to entering the studies amounted to 35.4 units packed per patient. In an uncontrolled trial oestrogen-progesterone therapy diminished significantly transfusion requirements from 3.4 units packed cells per patient per month to 0.1 units packed cells (p = 0.02). Haemorrhage ceased totally in 3 of 7 patients. A double-blind randomized placebo-controlled, cross-over trial shows that therapy with 0.050 mg ethinyloestradiol and 1 mg norethisterone is very effective in reducing transfusion requirements: 2.8 versus 11.2 units packed cells over a 6 month treatment period (p = 0.002). Only 3 of 13 patients treated with ethinyloestradiol and norethisterone required transfusions for persistent bleeding, while 12 of 13 patients in the placebo group had transfusion requirements (p = 0.001). After stopping hormonal therapy, no transfusions were needed for a mean of 10 months. This indicates a long-lasting effect of ethinyloestradiol and norethisterone on bleeding and transfusion requirements.(ABSTRACT TRUNCATED AT 250 WORDS)