Amyloidosis, Polyneuropathy
Conditions
Brief summary
To investigate to what extent chronic axonal length-dependent polyneuropathy (CAP) and/or small-fiber neuropathy (SFN) is part of early non-cardiac manifestations of wild-type TTR cardiac amyloidosis (wtTTR-CA). Consequently, explore whether this could ultimately lead to faster diagnosis and clinical outcome of wild-type TTR cardiac amyloidosis (wtTTR-CA).
Detailed description
Patients with chronic axonal length-dependent polyneuropathy (CAP) and/or small-fiber neuropathy (SFN) without well-defined cause will be recruited after a neurological standard routine work-up with NCS (Nerve conduction study) test, EMG, and Sudoscan®, previously performed at the neurology department of UZ-Brussel in normal clinical setting. All participants will be invited to the Neurology and Cardiology department for one visit on one day, for the following assessments: Following exams will be performed: * assessment of symptoms, severity, and duration of the polyneuropathy and the use of NTSS-6 and COMPASS31 score for mapping somatosensory and autonomic symptoms * evaluation of objective polyneuropathy signs, using following scales: mPND, NIS * Kansas City Cardiomyopathy Questionnaire (KCCQ) * Electrocardiogram (ECG) * Echocardiography The following retrospective data from the medical file will be analyzed: * assessment of medical history, medical treatment, and demographic data * assessment of laboratory results (and, if applicable, other exams) extracted from the medical file and previously performed in the context of polyneuropathy workup * assessment of previously performed NCV/EMG data and Sudoscan®, extracted from the medical file of the participants.
Interventions
DIAGNOSTIC_TESTECG + echocardiography
Electrocardiogram and echocardiography
Answering questionnaires about polyneuropathy symptoms (NTSS -6, COMPASS31, NIS, mPND) and also cardialogical symptoms (KCCQ-12).
Sponsors
Universitair Ziekenhuis Brussel
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
SCREENING
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
60 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Patients with chronic axonal length-dependent polyneuropathy (CAP) and/or small-fiber neuropathy (SFN) without well-defined etiology. * Age: \>= 60 years * Male and female gender * Written informed consent
Exclusion criteria
* Known cause of polyneuropathy * Other types of peripheral neuropathy than chronic axonal length-dependent polyneuropathy (CAP) and/or small-fiber neuropathy (SFN). * Patients younger than 60 years
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Primary Outcome: Evaluation of prevalence of wild-type cardiac amyloidosis in our CAP and/or SFN population without well known cause. | 36 months | Prevalence of cardiac amyloidosis by doing a cardiac screening for wild-type cardiac amyloidosis with performing an echocardiography. Echocardiography criteria: LV wall (\>12mm), left ventricular ejection fraction (%), Apical sparing pattern (y/n), diastolic dysfunction (y/n), left atrial volume (ml/m2). Left atrial volume in ml and LA volume in ml/m2, whereby m2 is the body surface area based on weight and height (Mosteller formula). |
| Primary Outcome: Evaluation of prevalence of wild-type cardiac amyloidosis in our CAP and/or SFN population without well known cause | 36 months | Prevalence of cardiac amyloidosis by doing a cardiac screening for wild-type cardiac amyloidosis with performing an ECG. ECG criteria: atrial fibrillation (y/n), QRS-duration, low voltage (y/n) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Secondary outcome: severity and evolution | 36 months | Considering the rather small study population and the rarity of wtTTR amyloidosis, statistical analyses cannot be used without massively overfitting the results, which will not be reproducible. The investigators consequently opt for descriptive statistics to compare the group of polyneuropathy patients with wt-Ca and those without wt-CA. Therefore a few parameters are examined. The first scale used therefore is the modified Polyneuropathy Disability score (mPND). This scale is a questionnaire with a few questions about the complaints of the polyneuropathy in the daily life. O means no complaints of the polyneuropathy in daily life, IV means a major impact on the daily life of the patient. |
| Secondary outcome: red flags that could increase the awareness of neurologists for wild-type TTR-cardiac amyloidosis | 36 months | The red flags for wild-type TTR cardiac amyloidosis are among others carpal tunnel syndrome (CTS), wich can be detected by performing an electromyography (EMG). |
Countries
Belgium
Contacts
Primary ContactVéronique Bissay, MD,Phd
Outcome results
None listed