Prevalence of Wild-type TTR Cardiac Amyloidosis in Patients With Polyneuropathy of Unknown Cause.

Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age60+
SponsorUniversitair Ziekenhuis Brussel

About this trial

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).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Single group

Treatments tested in this trial

  • ECG + echocardiography

    Diagnostic test

    Electrocardiogram and echocardiography

  • Answering questionnaires

    Other intervention

    Answering questionnaires about polyneuropathy symptoms (NTSS -6, COMPASS31, NIS, mPND) and also cardialogical symptoms (KCCQ-12).

Treatment groups

150 Participants
are divided into 1 treatment group
Group A: 1 arm including patients with CAP and/or SFNOther 2 interventions

Trial outcomes

Primary outcomes

1

Primary Outcome: Evaluation of prevalence of wild-type cardiac amyloidosis in our CAP and/or SFN population without well known cause.

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).

Time frame
36 months
2

Primary Outcome: Evaluation of prevalence of wild-type cardiac amyloidosis in our CAP and/or SFN population without well known cause

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)

Time frame
36 months
3

Primary Outcome: Evaluation of prevalence of wild-type cardiac amyloidosis in our CAP and/or SFN population without well known cause

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.

Time frame
36 months

Secondary outcomes

1

Secondary outcome: severity and evolution

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.

Time frame
36 months
2

Secondary outcome: severity and evolution

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 second scale used therefore is the Neuropathy Impairment Score (NIS).This scale is a questionnaire with a few questions about the complaints of the polyneuropathy in the daily life. The score is between 0 and 244. O means less complaints in the daily life, 244 means major impact in the daily life of the patient.

Time frame
36 months
3

Secondary outcome: severity and evolution

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 third scale used therefore is the Neuropathy Total Symptom Score - Health Care professional administered version (NTSS-6). This scale is a questionnaire with a few items about the symptoms of the polyneuropathy in the daily life (aching pain, burning pain, prickling sensation, numbness, lancinating pain and allodynia). Depending on the presence of the symptoms in time and strength, a score will show up. On each symptom you can score a maximum of 3,66. So the total score for this scale is 21,96 if the patients has major complaints in daily life.

Time frame
36 months
4

Secondary outcome: severity and evolution

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.

Time frame
36 months

Other outcomes

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