We hypothesize that structural changes in the eye muscles are specific to MG and are sensitive to treatment and therefore can be used in differential diagnosis and to predict treatment response.
ID
Bron
Verkorte titel
Aandoening
Myasthenia gravis, Graves' orbitopathy
Ondersteuning
Onderzoeksproduct en/of interventie
Uitkomstmaten
Primaire uitkomstmaten
(1) QMRI parameters (muscle fat fraction, muscle inflammation, muscle volume) which are hypothesized to differ between MG
patients and the healthy/disease controls for diagnostic value.
(2) For the second objective comparing recently diagnosed and chronic MG patients for exploring the pathophysiology
Achtergrond van het onderzoek
The auto-immune disease myasthenia gravis (MG) affects the neuromuscular junction (NMJ) and commonly starts with weakness of the extra-ocular muscles (EOM). In patients with pure EOM symptoms and no acetylcholine receptor (AChR) antibodies, diagnosis is difficult and time-consuming. This causes significant burden for patients and delays effective treatment.
Furthermore, treatment usually consists of immune suppressant medication, of which corticosteroids are the most commonly prescribed. Unfortunately, long term steroid use carries a considerable risk of unacceptable side effects. While immune suppressant treatment leads to a significant improvement in the majority of patients, 15% show only a moderate improvement or no improvement at all after treatment with corticosteroids.
Using magnetic resonance imaging (MRI), we have shown structural changes in the EOM of a small group of MG patients, which has not been described in literature before and was not expected based on the current knowledge of the pathophysiology. We hypothesize that these changes are specific to MG and are sensitive to treatment and therefore can be used in differential diagnosis and to predict treatment response. Therefore, we aim to develop a novel diagnostic paradigm for AChR negative MG and a predictor for treatment efficacy in all MG patients by systematically comparing qMRI parameters of the EOM in clearly defined clinical groups of MG patients to healthy and disease controls (other neuromuscular diseases and Graves’ orbitopathy (GO)) and over time.
Doel van het onderzoek
We hypothesize that structural changes in the eye muscles are specific to MG and are sensitive to treatment and therefore can be used in differential diagnosis and to predict treatment response.
Onderzoeksopzet
01-Jul-2022
Publiek
Wetenschappelijk
Belangrijkste voorwaarden om deel te mogen nemen (Inclusiecriteria)
Recently diagnosed MG patients
- Definitive diagnosis of MG defined as the presence of serum autoantibodies (anti—acetylcholine receptor [AChR],
anti-muscle specific tyrosine kinase [MUSK])
- Start of symptoms was less than a year ago
- No corticosteroid treatment received in the past year
- No TSH-receptor auto-antibodies, no laboratory signs of thyroid dysfunction (T4, TSH), Chronic MG patients
- Definitive diagnosis of MG, defined as described above
- Persisting symptoms of diplopia
- Start of symptoms was more than a year ago
- No TSH-receptor auto-antibodies, no laboratory signs of thyroid dysfunction (T4, TSH), Seronegative MG
patients
- Clinical diagnosis of MG with asymmetric, fluctuating and fatigable muscle weakness and at least one abnormal
neurophysiological test, indicative of neuromuscular dysfunction (repetitive nerve stimulation or single fiber EMG)
- No serum autoantibodies (anti—acetylcholine receptor [AChR], anti-muscle specific tyrosine kinase [MuSK])
- No TSH-receptor auto-antibodies, no laboratory signs of thyroid dysfunction (T4, TSH)
Healthy controls
- No symptoms of diplopia
- No ophthalmopathy
- No prior systemic treatment with corticosteroids, Patient controls: Graves' orbitopathy
- Definitive diagnosis of Graves' orbitopathy, Patient controls: Other neuromuscular disease
- Definitive diagnosis of a neuromuscular disease other than Myasthenia gravis
Belangrijkste redenen om niet deel te kunnen nemen (Exclusiecriteria)
- Subjects who are not legally capable
- Subjects under the age of 18
- Contraindications to MRI scanning, including:
o Claustrophobia
o Pregnancy
o Pacemakers and defibrillators
o Nerve stimulators
o Intracranial clips
o Metallic fragments
o Cochlear implants
o Ferromagnetic implants
o Hydrocephalus pump
o Permanent make-up
o Tattoos above the shoulders
o Piercings (unless they can be removed)
o Subjects who cannot keep their head still (eg. Tremor, Parkinson's disease)
o Severe physical restriction (completely wheelchair dependent)
❑ In the case of uncertainty about the MRI-contraindications, the MR-safety commission of the radiology
department will decide whether this subject can be included in the study or not.
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| Register | ID |
|---|---|
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| CCMO | NL68612.058.18 |
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