To assess the prevalence, severity and impact of arthropathy in patients with moderate or severe VWD and a history of documented DDAVP or clotting factor concentrate treatment for joint bleeds. To compare arthropathy in these patients to age, FVIII…
ID
Source
Brief title
Condition
- Coagulopathies and bleeding diatheses (excl thrombocytopenic)
- Joint disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Existence of arthropathy: the number and percentage of patients with
arthropathy among the different patient groups.
Severity of arthropathy: separate and cumulative scores of HJHS and Pettersson.
Impact of arthropathy on physical functioning and participation: time to
complete the figure 8 walking test, separate and cumulative scores of (Ped)HAL
and IPA questionnaires.
Impact on quality of life: separate and cumulative scores of D-AIMS2-affect and
MPQ-DLV questionnaires.
Secondary outcome
Influence of the severity of the coagulation defect on the existence, severity
and impact of arthropathy
Number and sites of affected joints
Quantitative use of desmopressin and coagulation factors in relation to the
existence of arthropathy
Prophylactic use of coagulation factors in relation to the existence of
arthropathy
Influence of age, BMI, quantitative use of desmopressin and coagulation
factors, surgical interventions and sports activities on the existence,
severity and impact of arthropathy
Background summary
Little is known about the severity and impact of arthropathy in patients with
severe and moderate severe von Willebrands disease (VWD).
Study objective
To assess the prevalence, severity and impact of arthropathy in patients with
moderate or severe VWD and a history of documented DDAVP or clotting factor
concentrate treatment for joint bleeds. To compare arthropathy in these
patients to age, FVIII and sex matched VWD patients without documented joint
bleeds and to age and FVIII matched patients with haemophilia A.
Study design
Cross-sectional multicenter cohort study coordinated from the Van
Creveldkliniek (VCK) of the University Medical Center Utrecht (UMCU).
Study burden and risks
This study will be the first to assess severity and impact of arthropathy in
patients with VWD. The total amount of time required is minimum 1 hour and 3
hours for the visit and measurements which will be combined with regular visits
if possible. Filling in the questionnaires will take approximately 60 minutes
of extra time for adult participants and 10 minutes for subjects <18 years. The
participating patients will not benefit directly from participation. However,
when arthropathy is found, the physiotherapist can give advice or refer
patients for further treatment on their request. Furthermore, better
understanding of arthropathy in WVD can lead to future preventive strategies
and improvement in information to young patients facing possible future
consequences of their disease. Risks imposed by participation are considered
negligible. However, because subjects<18 years can participate, there is a
minor increase over minimal risk.
Heidelberglaan 100
Utrecht 3584 CX
NL
Heidelberglaan 100
Utrecht 3584 CX
NL
Listed location countries
Age
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
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
In other registers
| Register | ID |
|---|---|
| CCMO | NL38989.041.12 |
| OMON | NL-OMON24768 |