Whole-body MRI in Gaucher disease; monitoring disease activity, early detection of complications and treatment decision-making
Completed
- Conditions
- Gaucher diseaseglucosylceramidosis10000546
- Registration Number
- NL-OMON44801
- Lead Sponsor
- Academisch Medisch Centrum
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 80
Inclusion Criteria
Age >18 years
Ability to provide informed consent
Patients: confirmed diagnosis Gaucher disease
Controls: resemblance in age and sex to matched patient
Exclusion Criteria
Age <18 years
Contra-indication MRI
Controls:
- known disease/abnormalities of liver, spleen or bone marrow. Or any of these in medical history.
- possible causes of iron overload
Study & Design
- Study Type
- Observational invasive
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The main study parameter is the difference in the measured ironlevels<br /><br>(expressed as R2*, a MRI based value correlating with ironconcentration (µmol/<br /><br>g)) between Gaucherpatients and healthy controls.</p><br>
- Secondary Outcome Measures
Name Time Method <p>- Comparison of ironmeasurements with standard care monitoring, that is liver-<br /><br>and spleenvolumes, QCSI and BMB-scores.<br /><br>- Relation ironmeasurements with biochemical GD markers<br /><br>- The value of extending the QCSI measurements, including tibia and fibula<br /><br>- Reproducibility of the whole-body MRI protocol</p><br>