Prevalence of Wild-type TTR Cardiac Amyloidosis in Patients With Polyneuropathy of Unknown Cause.
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: ECG + echocardiography, Answering questionnaires.
- Who it may be relevant to
- Registry conditions: Polyneuropathy, Amyloidosis. Basic parameters: from 60 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Belgium
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Prevalence of Wild-type TTR Cardiac Amyloidosis in Patients With Polyneuropathy of Unknown Cause: a Prospective Monocentric Study (CAP-TTR)
Overview
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 test ECG + echocardiography
Electrocardiogram and echocardiography - Other Answering questionnaires
Answering questionnaires about polyneuropathy symptoms (NTSS -6, COMPASS31, NIS, mPND) and also cardialogical symptoms (KCCQ-12).
Primary outcome measures
- Primary Outcome: Evaluation of prevalence of wild-type cardiac amyloidosis in our CAP and/or SFN population without well known cause. [Time frame: 36 months]
- Primary Outcome: Evaluation of prevalence of wild-type cardiac amyloidosis in our CAP and/or SFN population without well known cause [Time frame: 36 months]
- Primary Outcome: Evaluation of prevalence of wild-type cardiac amyloidosis in our CAP and/or SFN population without well known cause [Time frame: 36 months]
Secondary outcome measures (7)
- Secondary outcome: severity and evolution [Time frame: 36 months]
- Secondary outcome: severity and evolution [Time frame: 36 months]
- Secondary outcome: severity and evolution [Time frame: 36 months]
- Secondary outcome: severity and evolution [Time frame: 36 months]
- Secondary outcome: red flags that could increase the awareness of neurologists for wild-type TTR-cardiac amyloidosis [Time frame: 36 months]
- Secondary outcome: red flags that could increase the awareness of neurologists for wild-type TTR-cardiac amyloidosis [Time frame: 36 months]
- Secondary outcome: red flags that could increase the awareness of neurologists for wild-type TTR-cardiac amyloidosis [Time frame: 36 months]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Screening
Study locations
Belgium · 1 center
- UZ Brussel — Jette
Publications
- Russell A, Hahn C, Chhibber S, Korngut L, Fine NM. Utility of Neuropathy Screening for Wild-Type Transthyretin Amyloidosis Patients. Can J Neurol Sci. 2021 Sep;48(5):607-615. doi: 10.1017/cjn.2020.271. Epub 2020 Dec 21. PMID 33342448
- Kleefeld F, Scherret E, Knebel F, Messroghli D, Heidecker B, Wetz C, Schatka I, Barzen G, Tschope C, Amthauer H, Hahn K. Same same, but different? The neurological presentation of wildtype transthyretin (ATTRwt) amyloidosis. Amyloid. 2022 Jun;29(2):92-101. doi: 10.1080/13506129.2021.2014448. Epub 2022 Jan 7. PMID 34994254
- Papagianni A, Ihne S, Zeller D, Morbach C, Uceyler N, Sommer C. Clinical and apparative investigation of large and small nerve fiber impairment in mixed cohort of ATTR-amyloidosis: impact on patient management and new insights in wild-type. Amyloid. 2022 Mar;29(1):14-22. doi: 10.1080/13506129.2021.1976751. Epub 2021 Oct 11. PMID 34632904
- Barroso FA, Coelho T, Dispenzieri A, Conceicao I, Waddington-Cruz M, Wixner J, Maurer MS, Rapezzi C, Plante-Bordeneuve V, Kristen AV, Gonzalez-Duarte A, Chapman D, Stewart M, Amass L; THAOS investigators. Characteristics of patients with autonomic dysfunction in the Transthyretin Amyloidosis Outcomes Survey (THAOS). Amyloid. 2022 Sep;29(3):175-183. doi: 10.1080/13506129.2022.2043270. Epub 2022 Apr PMID 35451899
- Kharoubi M, Roche F, Bezard M, Hupin D, Silva S, Oghina S, Chalard C, Zaroui A, Galat A, Guendouz S, Canoui-Poitrine F, Hittinger L, Teiger E, Lefaucheur JP, Damy T. Prevalence and prognostic value of autonomic neuropathy assessed by Sudoscan(R) in transthyretin wild-type cardiac amyloidosis. ESC Heart Fail. 2021 Apr;8(2):1656-1665. doi: 10.1002/ehf2.13131. Epub 2020 Dec 22. PMID 33354901
- Wajnsztajn Yungher F, Kim A, Boehme A, Kleyman I, Weimer LH, Maurer MS, Brannagan TH 3rd. Peripheral neuropathy symptoms in wild type transthyretin amyloidosis. J Peripher Nerv Syst. 2020 Sep;25(3):265-272. doi: 10.1111/jns.12403. Epub 2020 Jul 27. PMID 32627282
- Zis P, Sarrigiannis PG, Rao DG, Hewamadduma C, Hadjivassiliou M. Chronic idiopathic axonal polyneuropathy: a systematic review. J Neurol. 2016 Oct;263(10):1903-10. doi: 10.1007/s00415-016-8082-7. Epub 2016 Mar 9. PMID 26961897
- Li Y. Axonal Sensorimotor Polyneuropathies. Continuum (Minneap Minn). 2017 Oct;23(5, Peripheral Nerve and Motor Neuron Disorders):1378-1393. doi: 10.1212/CON.0000000000000514. PMID 28968367
Identifiers
NCT: NCT05950867 · EC-2023-115