Arthropathy in patients with congenital severe and moderate severe von Willebrand disease

Completed

Conditions: athropathy
joint damage
10064477
10023213

Registration Number: NL-OMON39851

Lead Sponsor: niversitair Medisch Centrum Utrecht

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 100

Inclusion Criteria

Patients with moderate or severe VWD who participated in the *Willebrand in Nederland* (WiN) study and who reported treatment with coagulation factor or desmopressin for 1 or more joint bleeds (patients)
Patients with moderate or severe VWD who participated in the *Willebrand in Nederland* (WiN) study and did not report treatment with coagulation factor or desmopressine for 1 or more joint bleeds (controls)
Patients with moderate or severe VWD who are currently treated at a haemophilia treatment centre in the Netherlands with or without a history of coagulation factor or desmopressin for 1 or more joint bleeds
Comprehension of the Dutch written and spoken language

Exclusion Criteria

Inability of the patient or the patients parents to give informed consent
Active joint pathology (i.e. recent episode of joint haemorrhage)
Restricted motion of an ankle, knee or elbow joint for another medical reason
No medical file available
Age 4 years or younger

Study & Design

Study Type: Observational non invasive

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Existence of arthropathy: the number and percentage of patients with<br /><br>arthropathy among the different patient groups.<br /><br>Severity of arthropathy: separate and cumulative scores of HJHS and Pettersson.<br /><br>Impact of arthropathy on physical functioning and participation: time to<br /><br>complete the figure 8 walking test, separate and cumulative scores of (Ped)HAL<br /><br>and IPA questionnaires.<br /><br>Impact on quality of life: separate and cumulative scores of D-AIMS2-affect and<br /><br>MPQ-DLV questionnaires.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Influence of the severity of the coagulation defect on the existence, severity<br /><br>and impact of arthropathy<br /><br>Number and sites of affected joints<br /><br>Quantitative use of desmopressin and coagulation factors in relation to the<br /><br>existence of arthropathy<br /><br>Prophylactic use of coagulation factors in relation to the existence of<br /><br>arthropathy<br /><br><br /><br>Influence of age, BMI, quantitative use of desmopressin and coagulation<br /><br>factors, surgical interventions and sports activities on the existence,<br /><br>severity and impact of arthropathy</p><br>