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

Официальное название

Prevalence of Wild-type TTR Cardiac Amyloidosis in Patients With Polyneuropathy of Unknown Cause: a Prospective Monocentric Study (CAP-TTR)

Обзор

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).

Подробное описание

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.

Вмешательства

• Диагностический тест ECG + echocardiography
  Electrocardiogram and echocardiography
• Другое Answering questionnaires
  Answering questionnaires about polyneuropathy symptoms (NTSS -6, COMPASS31, NIS, mPND) and also cardialogical symptoms (KCCQ-12).

Первичные конечные точки

• Primary Outcome: Evaluation of prevalence of wild-type cardiac amyloidosis in our CAP and/or SFN population without well known cause. [Срок оценки: 36 months]
• Primary Outcome: Evaluation of prevalence of wild-type cardiac amyloidosis in our CAP and/or SFN population without well known cause [Срок оценки: 36 months]
• Primary Outcome: Evaluation of prevalence of wild-type cardiac amyloidosis in our CAP and/or SFN population without well known cause [Срок оценки: 36 months]

Вторичные конечные точки (7)

• Secondary outcome: severity and evolution [Срок оценки: 36 months]
• Secondary outcome: severity and evolution [Срок оценки: 36 months]
• Secondary outcome: severity and evolution [Срок оценки: 36 months]
• Secondary outcome: severity and evolution [Срок оценки: 36 months]
• Secondary outcome: red flags that could increase the awareness of neurologists for wild-type TTR-cardiac amyloidosis [Срок оценки: 36 months]
• Secondary outcome: red flags that could increase the awareness of neurologists for wild-type TTR-cardiac amyloidosis [Срок оценки: 36 months]
• Secondary outcome: red flags that could increase the awareness of neurologists for wild-type TTR-cardiac amyloidosis [Срок оценки: 36 months]

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Скрининг

Центры проведения

Бельгия · 1 центр

• UZ Brussel — Jette

Публикации

• 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

Идентификаторы

NCT: NCT05950867 · EC-2023-115

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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