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Liver and Coagulation Disorders in Cardiac Transthyretin Amyloidosis (LICA2025)

2026年8月10日 更新者：Gianluca Di Bella、University of Messina

Liver and Coagulation Disorders in Cardiac Transthyretin Amyloidosis (ATTR-CA): New Horizons in Disease Staging and Follow-Up

Transthyretin cardiac amyloidosis (ATTR-CA) is a progressive infiltrative cardiomyopathy caused by the deposition of misfolded transthyretin protein within the myocardium. Current disease staging and follow-up strategies mainly rely on cardiac biomarkers and renal function; however, the systemic nature of ATTR suggests that additional organ involvement may provide valuable prognostic information.

The purpose of this prospective observational study is to investigate liver dysfunction and coagulation abnormalities in patients with wild-type or hereditary ATTR-CA and to evaluate their potential role as novel markers of disease severity and progression. Patients with ATTR-CA will be compared with an age-matched control population with non-amyloid hypertrophic cardiomyopathy.

Clinical, laboratory, echocardiographic, hepatic ultrasound, liver stiffness, and coagulation parameters will be assessed at baseline and during follow-up. The study will also evaluate changes in these parameters after 6 and 12 months of treatment with tafamidis.

The results may improve the understanding of cardio-hepatic interactions in ATTR-CA and identify new tools for disease staging and longitudinal monitoring.

調査の概要

状態

募集

条件

詳細な説明

Transthyretin cardiac amyloidosis (ATTR-CA) is an increasingly recognized cause of heart failure and left ventricular hypertrophy in older adults. Although several prognostic models have been developed for ATTR-CA, most currently available staging systems are based primarily on cardiac biomarkers and renal function. These approaches may not fully capture the systemic nature of the disease.

Emerging evidence suggests that liver dysfunction and coagulation abnormalities may represent underexplored manifestations of ATTR-CA. Liver involvement may result from direct amyloid deposition, chronic venous congestion related to heart failure, or a combination of both mechanisms. Similarly, alterations in coagulation pathways may reflect hepatic dysfunction and systemic disease burden.

LICA2025 is a single-center, prospective, observational study designed to evaluate biochemical and instrumental markers of liver function and coagulation in patients with wild-type or hereditary ATTR-CA followed at the University Hospital G. Martino of Messina, Italy. A control population with non-amyloid hypertrophic cardiomyopathy will be enrolled for comparison.

The primary objective is to compare liver and coagulation parameters between ATTR-CA patients and controls. Secondary objectives include evaluating the relationship between hepatic/coagulation abnormalities and cardiac disease severity, assessing changes after 6±1 and 12±1 months of tafamidis therapy, and exploring potential interactions between liver dysfunction and coagulation disturbances.

Participants will undergo clinical evaluation, laboratory testing, electrocardiography, transthoracic echocardiography, hepatic ultrasound, liver elastography (FibroScan), and coagulation assessment according to the study protocol. Follow-up evaluations will be performed at baseline, 6 months, and 12 months.

The study aims to identify novel biomarkers and imaging parameters that may improve disease staging, risk stratification, and longitudinal monitoring in transthyretin cardiac amyloidosis.

研究の種類

観察的

入学 (推定)

70

連絡先と場所

このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。

研究連絡先

研究場所

  • イタリア
    • Sicily
      • Messina、Sicily、イタリア、98124
        • 募集
        • AOU Policlinico G. Martino, UOC Cardiologia con UTIC
        • コンタクト：
        • 副調査官：
          • Roberto Licordari, MD, PhD
        • 主任研究者：
          • Luigi Colarusso, MD, PhD Candidate

参加基準

研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。

適格基準

就学可能な年齢

  • 大人
  • 高齢者

健康ボランティアの受け入れ

いいえ

サンプリング方法

非確率サンプル

調査対象母集団

Adult patients with wild-type or hereditary transthyretin cardiac amyloidosis (ATTR-CA) followed at the University Hospital G. Martino, Messina, Italy, and an age-matched control population with non-amyloid hypertrophic phenotype cardiomyopathy.

説明

Inclusion Criteria:

  • Written informed consent obtained prior to study participation.
  • Diagnosis of wild-type or hereditary transthyretin cardiac amyloidosis (ATTR-CA) according to current European recommendations.
  • Ability to comply with study procedures and follow-up visits.

Exclusion Criteria:

  • Age younger than 18 years.
  • Severe liver dysfunction due to causes other than amyloidosis.
  • Inability to comply with study procedures because of language barriers, cognitive impairment, or severe psychiatric disorders.
  • Comorbidities associated with life expectancy less than 12 months.
  • Active alcohol or substance abuse.
  • For coagulation analyses: congenital coagulation disorders, thrombotic disorders, active malignancy, or sepsis.
  • Pregnancy or breastfeeding.

研究計画

このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。

研究はどのように設計されていますか？

デザインの詳細

コホートと介入

グループ/コホート
ATTR-CA Patients
Patients with wild-type or hereditary transthyretin cardiac amyloidosis (ATTR-CA) diagnosed according to current European recommendations and followed at the University Hospital G. Martino, Messina. Participants will undergo clinical, laboratory, echocardiographic, hepatic ultrasound, liver elastography, and coagulation assessments at baseline and during follow-up.
Hypertrophic Phenotype Controls
Age-matched patients with non-amyloid hypertrophic phenotype cardiomyopathy serving as a control population. Participants will undergo the same clinical, laboratory, echocardiographic, hepatic, and coagulation evaluations as the ATTR-CA group.

この研究は何を測定していますか？

主要な結果の測定

結果測定
メジャーの説明
時間枠
Liver Stiffness Measured by Transient Elastography (FibroScan)
時間枠：Baseline, 6 Months, 12 Months
Liver stiffness expressed in kilopascals (kPa) measured by transient elastography (FibroScan) in ATTR-CA patients and controls.
Baseline, 6 Months, 12 Months
Controlled Attenuation Parameter (CAP) Measured by FibroScan
時間枠：Baseline, 6 months, 12 months
Controlled attenuation parameter (CAP) measured by FibroScan as an instrumental measure of hepatic steatosis, expressed in decibels per meter (dB/m), compared between patients with ATTR-CA and controls.
Baseline, 6 months, 12 months
Serum Aspartate Aminotransferase (AST/GOT)
時間枠：Baseline, 6 months, 12 months
Serum AST/GOT concentration (U/L) measured by routine laboratory testing, compared between patients with ATTR-CA and controls.
Baseline, 6 months, 12 months
Serum Alanine Aminotransferase (ALT/GPT)
時間枠：baseline, 6 months, 12 months
Serum ALT/GPT concentration (U/L) measured by routine laboratory testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Serum Gamma-Glutamyl Transferase (GGT)
時間枠：baseline, 6 months, 12 months
Serum gamma-glutamyl transferase concentration (U/L) measured by routine laboratory testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Serum Alkaline Phosphatase (ALP)
時間枠：baseline, 6 months, 12 months
Serum alkaline phosphatase concentration (U/L) measured by routine laboratory testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Serum Bile Acids
時間枠：baseline, 6 months, 12 months
Serum bile acid concentration (mcmol/L) measured by laboratory testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Total Bilirubin (mg/dL)
時間枠：baseline, 6 months, 12 months
Serum total bilirubin concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Direct Bilirubin (mg/dL)
時間枠：baseline, 6 months, 12 months
Serum direct bilirubin concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Serum Albumin (g/dL)
時間枠：baseline, 6 months, 12 months
Serum albumin concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Serum Gamma Globulins (g/dL)
時間枠：baseline, 6 months, 12 months
Serum gamma-globulin concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Serum Immunoglobulin M (IgM) (g/L)
時間枠：baseline, 6 months, 12 months
Serum IgM concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Serum Ferritin (ng/mL)
時間枠：baseline, 6 months, 12 months
Serum ferritin concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Anti-Mitochondrial Antibodies
時間枠：baseline, 6 months, 12 months
U/ml
baseline, 6 months, 12 months
Fibrosis-4 (FIB-4) Index
時間枠：baseline, 6 months, 12 months
FIB-4 index calculated from age, AST, ALT, and platelet count, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Portal Vein Diameter Assessed by Liver Ultrasound
時間枠：baseline, 6 months, 12 months
Portal vein diameter (mm) measured by liver ultrasound, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Portal Vein Flow Velocity Assessed by Doppler Ultrasound (cm/s)
時間枠：baseline, 6 months, 12 months
Portal vein blood flow velocity measured by Doppler ultrasound, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Spleen Diameter Assessed by Ultrasound (mm)
時間枠：baseline, 6 months, 12 months
Spleen diameter measured by abdominal ultrasound, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Prothrombin Time (PT) (s)
時間枠：baseline, 6 months, 12 months
Prothrombin time measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Activated Partial Thromboplastin Time (aPTT) (s)
時間枠：baseline, 6 months, 12 months
Activated partial thromboplastin time measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
International Normalized Ratio (INR)
時間枠：baseline, 6 months, 12 months
International normalized ratio (INR) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Plasma Fibrinogen
時間枠：baseline, 6 months, 12 months
Plasma fibrinogen concentration (g/L) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Fibrin/Fibrinogen Degradation Products
時間枠：Baseline, 6 months, 12 months
Fibrin/fibrinogen degradation products concentration (mcg/Lm) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
Baseline, 6 months, 12 months
D-Dimer
時間枠：baseline, 6 months, 12 months
D-dimer concentration (ng/mL) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Alpha-2-Antiplasmin
時間枠：baseline, 6 months, 12 months
Alpha-2-antiplasmin concentration (UI/ml) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Antithrombin
時間枠：baseline, 6 months, 12 months
Antithrombin concentration (IU) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Plasminogen
時間枠：baseline, 6 months, 12 months
Plasminogen concentration (IU/ml) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Thrombin-Antithrombin Complex
時間枠：baseline, 6 months, 12 months
Plasma fibrinogen concentration (ng/mL) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Plasmin-Alpha-2-Antiplasmin Complex
時間枠：baseline, 6 months, 12 months
Plasmin-Alpha-2-Antiplasmin Complex concentration (ng/mL) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Prothrombin Fragment 1+2
時間枠：baseline, 6 months, 12 months
Prothrombin Fragment 1+2 concentration (pmol/L) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Coagulation Factor X
時間枠：baseline, 6 months, 12 months
Coagulation Factor X concentration (IU) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
von Willebrand Factor Antigen
時間枠：baseline, 6 months, 12 months
von Willebrand Factor Antigen concentration (IU/mL) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Plasminogen Activator Inhibitor-1 (PAI-1)
時間枠：baseline, 6 months, 12 months
Plasminogen Activator Inhibitor-1 concentration (U/mL) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months

二次結果の測定

結果測定
メジャーの説明
時間枠
Correlation Between Liver Stiffness (kPa) and NT-proBNP (ng/L)
時間枠：Baseline
Correlation between liver stiffness measured by FibroScan (kPa) and plasma NT-proBNP concentration (ng/L) in patients with ATTR-CA. Correlation will be assessed using Pearson or Spearman correlation coefficient, as appropriate.
Baseline
Correlation Between Liver Stiffness and Interventricular Septal Thickness (mm)
時間枠：Baseline
Correlation between liver stiffness measured by FibroScan (kPa) and interventricular septal thickness expressed in mm measured by transthoracic echocardiography in patients with ATTR-CA. Correlation will be assessed using Pearson or Spearman correlation coefficient, as appropriate.
Baseline
Correlation Between Liver Stiffness (kPa) and Left Ventricular Global Longitudinal Strain (GLS %)
時間枠：Baseline
Correlation between liver stiffness measured by FibroScan (kPa) and Global Longitudinal Strain (expressed in %) measured by transthoracic echocardiography in patients with ATTR-CA. Correlation will be assessed using Pearson or Spearman correlation coefficient, as appropriate.
Baseline
Correlation Between Prothrombin Fragment 1+2 (pmol/L) and NT-proBNP (ng/L)
時間枠：Baseline
Correlation between Prothrombin Fragment 1+2 (pmol/L) and NT-proBNP (ng/L) measured by standard laboratory testing in patients with ATTR-CA. Correlation will be assessed using Pearson or Spearman correlation coefficient, as appropriate.
Baseline
Change in Liver Stiffness During Disease-Modifying Treatment
時間枠：Baseline, 6±1 months, and 12±1 months
Change in liver stiffness measured by FibroScan and expressed in kPa from baseline to 6±1 months and 12±1 months after treatment initiation.
Baseline, 6±1 months, and 12±1 months
Change in Controlled Attenuation Parameter During Disease-Modifying Treatment
時間枠：Baseline, 6±1 months, and 12±1 months
Change in controlled attenuation parameter (CAP) measured by FibroScan and expressed in dB/m from baseline to 6±1 months and 12±1 months after treatment initiation.
Baseline, 6±1 months, and 12±1 months

その他の成果指標

結果測定
メジャーの説明
時間枠
Correlation Between Liver Stiffness and Prothrombin Fragment 1+2
時間枠：Baseline
Correlation between liver stiffness measured by transient elastography (FibroScan), expressed in kPa, and plasma prothrombin fragment 1+2 concentration in patients with ATTR-CA. Correlation will be assessed using Pearson or Spearman correlation coefficient, as appropriate.
Baseline
Correlation Between FIB-4 Index and Prothrombin Fragment 1+2
時間枠：Baseline
Correlation between FIB-4 and plasma prothrombin fragment 1+2 concentration in patients with ATTR-CA. Correlation will be assessed using Pearson or Spearman correlation coefficient, as appropriate.
Baseline
Correlation Between Liver Stiffness and Fibrinogen
時間枠：Baseline
Correlation between liver stiffness measured by transient elastography (FibroScan), expressed in kPa, and plasma fibrinogen concentration in patients with ATTR-CA. Correlation will be assessed using Pearson or Spearman correlation coefficient, as appropriate.
Baseline

協力者と研究者

ここでは、この調査に関係する人々や組織を見つけることができます。

スポンサー

University of Messina

協力者

Azienda Ospedaliera Universitaria Policlinico "G. Martino"

捜査官

  • スタディチェア：Gianluca Di Bella, MD, PhD、University of Messina and AOU Policlinico G. Martino
  • 主任研究者：Luigi Colarusso, MD, PhD Candidate、AOU Policlinico G. Martino

研究記録日

これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。

主要日程の研究

研究開始 (実際)

2026年1月30日

一次修了 (推定)

2028年1月1日

研究の完了 (推定)

2028年1月1日

試験登録日

最初に提出

2026年6月12日

QC基準を満たした最初の提出物

2026年8月10日

最初の投稿 (実際)

2026年8月14日

学習記録の更新

投稿された最後の更新 (実際)

2026年8月14日

QC基準を満たした最後の更新が送信されました

2026年8月10日

最終確認日

2026年6月1日

詳しくは

本研究に関する用語

キーワード

追加の関連 MeSH 用語

その他の研究ID番号

  • CEL Messina Prot. 28-25
  • Delibera n. 63/2026 (その他の識別子：AOU Policlinico G. Martino)

個々の参加者データ (IPD) の計画

個々の参加者データ (IPD) を共有する予定はありますか?

未定

IPD プランの説明

The decision regarding IPD sharing has not yet been finalized. Any future data sharing will be considered in accordance with applicable ethical approvals, participant consent, institutional policies, and data protection regulations.

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