Primary Objective: (diagnostic)• Does MRI detect structural changes of haemophilic arthropathy in knees and ankles undetected by the Pettersson score and HJHS? (Research question 1 a & b)Secondary Objective(s): (prognostic)• Can MRI predict loss…
ID
Source
Brief title
Condition
- Coagulopathies and bleeding diatheses (excl thrombocytopenic)
- Joint disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Determinant: MRI findings according to the Lundin additive Score.
Outcome parameters:
Diagnostic study: HJHS score (T=0)
Pettersson score (T=0)
Prognostic study: delta HJHS score (T=2.5 and T=5 years)
delta Pettersson score (T=5 years)
Secondary outcome
The association of MRI with delta HJHS after 2.5 and 5 years' interval will be
adjusted for baseline HJHS score, number of joint bleeds during the observation
interval, physical activity, and age.
The association of MRI with delta Pettersson score after 5 years' interval will
be adjusted for baseline Pettersson score, number of joint bleeds during the
observation interval, physical activity, and bone age.
Background summary
Repeated joint bleeds, eventually leading to crippling arthropathy, are the
hallmark of haemophilia. Traditionally, outcome assessment in haemophilia has
been focused on structural assessment of the six most frequently affected
joints: elbows, knees, and ankles. Since the 1980s, two haemophilia specific
tools have been available: the radiological Pettersson score and the clinical
joint score (or orthopaedic joint score). Both scoring systems have been
adapted by the World Federation of Haemophilia, and have been widely used, in
spite of their limitations. Recently, several new tools for outcome assessment
in haemophilia have been developed. As result of the efforts of the
International Prophylaxis Study Group (IPSG) a scoring system for structural
assessment using MRI (Lundin additive score), was developed and the orthopaedic
joint score was adapted into the HJHS (haemophilia joint health score) to
assess more subtle joint damage. Except for a recent RCT comparing prophylactic
to on demand treatment in young children with severe haemophilia (follow-up
until age 6 yrs), MRI has been used in older patients to diagnose synovitis,
rather than evaluate prophylactic treatment. Hence we do not know if our
teenage patients with a history of a few bleeds, but very little or no
arthropathy on X-ray and good clinical function, will have structural changes
on MRI.
In order to study the value of this instrument in relation to the other outcome
parameters and its ability to pick up early changes, a pilot study will be
performed among a subset of adolescents participating in two international
projects on severe and moderate haemophilia. As the power of a pilot study is
limited by definition, the results on MRI will only begin to address
correlation and potential discriminative value in comparison with other outcome
instruments and its ability to predict future changes.
Controls will be included to distinguish haemophilia-related abnormalities from
sports-related abnormalities.
Study objective
Primary Objective: (diagnostic)
• Does MRI detect structural changes of haemophilic arthropathy in knees and
ankles undetected by the Pettersson score and HJHS? (Research question 1 a & b)
Secondary Objective(s): (prognostic)
• Can MRI predict loss of function (measured by HJHS) in 2,5 and 5 years*
time? (Research question 2 a & b)
• Can MRI predict X-ray changes (measured by Pettersson score) in 5 years*
time? (Research question 3)
Study design
Diagnostic: cross-sectional within cohorts from other projects.
Prospective part: follow-up will be 5 years, using routine data.
Study burden and risks
Participants will undergo one MRI of 4 joints (4x 15 min in the MRI scan) and
an X ray examination of the wrist (5 min). Remaining measurements will be
collected from available data. The risk for participants is considered minimal.
The impact of MRI studies is expected to be most pronounced and clinically most
relevant in young adults and adolescents (aged 12-26): if the MRI findings
warrant intensification of treatment to further reduce the number of bleeds,
younger patients are expected to benefit more than older patients. Treatment
will not change during the study unless aforementioned unexpected significant
MRI changes warrant intensification of prophylactic treatment. This will not
affect the analyses, as damage is related to bleeds only.
Controls will undergo one MRI of 4 joints (4x 15 min in the MRI scan), as well
as a standardised physical examination (15-20 min) and complete two short
questionnaires (10-15 min). They are not expected to benefit from or be harmed
by participation in the study.
Heidelberglaan 100
Utrecht 3584 CX
NL
Heidelberglaan 100
Utrecht 3584 CX
NL
Listed location countries
Age
Inclusion criteria
• Haemophilia A or B
• Severe (<1% FVIII/IX activity) (n=20) or moderate haemophilia (1-5% FVIII/IX activity) (n=10).
• 12-26 years of age
• Recent Pettersson scores (within two years of assessment) available
• maximum one knee or ankle with a score above 3 points
• minimal two ankles or knees with a score of 0 points
• entry clinic before age 4
Controls:
• male, 18-26 years of age
• participating in regular sports (1-3x/wk )
Exclusion criteria
• History of inhibitors
• No access to standard care since birth (e.g. immigration from other
country)
• Last Pettersson score more than 2 years before assessment
• More than one knee or ankle with a Pettersson score above 3 points
• Less than two knees or ankles with a Pettersson score of 0 points
• Extensive bleeding in knees or ankles 2/3 weeks before assessment
Controls
• contraindication for MRI assessment
• knee/ankle injuries within 8 weeks prior to MRI
* sports participation less than once weekly or more than 3 times weekly
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL20590.041.08 |