Prevalence of Wild-type TTR Cardiac Amyloidosis in Patients With Polyneuropathy of Unknown Cause.
- Conditions
- PolyneuropathyAmyloidosis
- Interventions
- Diagnostic Test: ECG + echocardiographyOther: Answering questionnaires
- Registration Number
- NCT05950867
- Lead Sponsor
- Universitair Ziekenhuis Brussel
- 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 150
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description 1 arm including patients with CAP and/or SFN Answering questionnaires 1 arm including patients with chronic axonal length-dependent polyneuropathy and/or small-fiber neuropathy. All participants will be screened with ECG and echocardiography. All participants will be asked to complete questionnaires about there polyneuropathy and cardiological symptoms. 1 arm including patients with CAP and/or SFN ECG + echocardiography 1 arm including patients with chronic axonal length-dependent polyneuropathy and/or small-fiber neuropathy. All participants will be screened with ECG and echocardiography. All participants will be asked to complete questionnaires about there polyneuropathy and cardiological symptoms.
- Primary Outcome Measures
Name Time Method 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 a questionnaire (Kansas City Cardiomyopathy Questionnaire).
KCCQ-12 has 4 domains (Physical Limitation Score, Symptom Frequency Score, Quality of Life Score, Social Limitation Score) and one Summary Score. Scores are scaled 0-100, where 0 denotes the lowest reportable health status and 100 the highest.
This descriptive score will be used as a functional parameter without a cut-off.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).
- Secondary Outcome Measures
Name Time Method 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 fourth scale used therefore is the Composite Autonomic Symptom score (COMPASS-31). This scale is a questionnaire with a 31 questions about the complaints in the daily life and also if there complaints/symptoms on the autonomic system. A few questions are asked about different domains (orthostatic intolerance, vasomotor, secretomotor, gastrointestinal system, bladder, pupillomotor).The total score is minimal 0 and maximal 75 raw counted (100 weighted).Each domain had a weighting factor to be count depending on the answers 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 trigger finger.
Trial Locations
- Locations (1)
UZ Brussel
🇧🇪Jette, Belgium Capital City, Belgium