Efficacy, safety and pharmaco-economic assessment of secondary long term prophylaxis with highly purified, standardized, doubly virus inactivated FVIII/VWF concentrates in patients with severe, inherited VWD and frequent bleedings - PRO.WI

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 24

Inclusion Criteria

- Documented unresponsiveness to DDAVP. All the patients, excluding type 3 and type 2B, should have been exposed to a documented infusion trial with DDAVP. Responsiveness to DDAVP is defined as an increase in plasma FVIII C and VWF RCo levels of at least three fold over baseline and in absolute reaching at least 30 IU/dl for both values and a BT of 12 minutes or less after 2 hours from administration of 0,3 mcg/kg of DDAVP OR contraindication to DDAVP namely type 2B VWD patients or other patients who display significant side effects to DDAVP - Frequent, spontaneous bleedings i.e. at least 5 episodes in whatever anatomical site in the last 12 months, severe enough to require treatment with FVIII/VWF concentrates Note that prolonged or excess bleeding during menses is not considered among inclusion criteria in this study OR Recurrent spontaneous bleedings, severe enough to require treatment with FVIII/VWF concentrates of the following types 61607; Epistaxes at least 5 episodes in the last 12 months 61607; Haemarthroses at least 3 episodes at the same joint in the last 12 months 61607; Gastrointestinal bleeding at least 2 episodes in the last 12 months, with a drop 61619; 2 g/dl of haemoglobin in 24-48 hours due to unexplained reason or in association with underlying gastrointestinal angiodysplasia - Willingness to participate in the study, expressed by signed, written informed consent
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- a life expectancy 1 year - presence of allo-antibodies to VWF or FVIII - acquired Von Willebrand syndrome AVWS - co-morbidity with other haemorrhagic diathesis, excluded those linked to VWD as the complication of treatment for instance thrombocytopenia in Type 2B VWD - advanced liver cirrhosis with 61607; cirrhosis related coagulation abnormalities thrombocytopenia, defined as platelet count 50.000/mm3, INR 1,7, prolonged prothrombin time 4 seconds versus normal 61607; portal hypertension with history of variceal bleeding - pregnancy and lactation - any known need for invasive procedures or elective surgery scheduled in the following 3 months recruitment in these cases should be postponed - proven co-morbidity for other causes of gastrointestinal bleeding not related to the studied disease as drug induced haemorrhagic gastropathy, upper GI tract ulcers or cancer, or operable conditions, e.g. haemorrhoids with the exception of concomitant angiodysplasia which meets the inclusion criteria - gastrointestinal bleeding due to trauma, invasive diagnostic or surgical procedures - concomitant autoimmune anaemia and/or autoimmune thrombocytopenia

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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