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.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
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.
Primary outcome measure(s)
Liver Stiffness Measured by Transient Elastography (FibroScan) — Baseline, 6 Months, 12 Months Liver stiffness expressed in kilopascals (kPa) measured by transient elastography (FibroScan) in ATTR-CA patients and controls.
Controlled Attenuation Parameter (CAP) Measured by FibroScan — 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.
Serum Aspartate Aminotransferase (AST/GOT) — Baseline, 6 months, 12 months Serum AST/GOT concentration (U/L) measured by routine laboratory testing, compared between patients with ATTR-CA and controls.
Serum Alanine Aminotransferase (ALT/GPT) — baseline, 6 months, 12 months Serum ALT/GPT concentration (U/L) measured by routine laboratory testing, compared between patients with ATTR-CA and controls.
Serum Gamma-Glutamyl Transferase (GGT) — 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.
Serum Alkaline Phosphatase (ALP) — baseline, 6 months, 12 months Serum alkaline phosphatase concentration (U/L) measured by routine laboratory testing, compared between patients with ATTR-CA and controls.
Serum Bile Acids — baseline, 6 months, 12 months Serum bile acid concentration (mcmol/L) measured by laboratory testing, compared between patients with ATTR-CA and controls.
Total Bilirubin (mg/dL) — baseline, 6 months, 12 months Serum total bilirubin concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
Direct Bilirubin (mg/dL) — baseline, 6 months, 12 months Serum direct bilirubin concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
Serum Albumin (g/dL) — baseline, 6 months, 12 months Serum albumin concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
Serum Gamma Globulins (g/dL) — baseline, 6 months, 12 months Serum gamma-globulin concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
Serum Immunoglobulin M (IgM) (g/L) — baseline, 6 months, 12 months Serum IgM concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
Serum Ferritin (ng/mL) — baseline, 6 months, 12 months Serum ferritin concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
Fibrosis-4 (FIB-4) Index — baseline, 6 months, 12 months FIB-4 index calculated from age, AST, ALT, and platelet count, compared between patients with ATTR-CA and controls.
Portal Vein Diameter Assessed by Liver Ultrasound — baseline, 6 months, 12 months Portal vein diameter (mm) measured by liver ultrasound, compared between patients with ATTR-CA and controls.
Portal Vein Flow Velocity Assessed by Doppler Ultrasound (cm/s) — baseline, 6 months, 12 months Portal vein blood flow velocity measured by Doppler ultrasound, compared between patients with ATTR-CA and controls.
Spleen Diameter Assessed by Ultrasound (mm) — baseline, 6 months, 12 months Spleen diameter measured by abdominal ultrasound, compared between patients with ATTR-CA and controls.
Prothrombin Time (PT) (s) — baseline, 6 months, 12 months Prothrombin time measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
Activated Partial Thromboplastin Time (aPTT) (s) — baseline, 6 months, 12 months Activated partial thromboplastin time measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
International Normalized Ratio (INR) — baseline, 6 months, 12 months International normalized ratio (INR) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
Plasma Fibrinogen — baseline, 6 months, 12 months Plasma fibrinogen concentration (g/L) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
Fibrin/Fibrinogen Degradation Products — 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.
D-Dimer — baseline, 6 months, 12 months D-dimer concentration (ng/mL) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
Alpha-2-Antiplasmin — 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.
Antithrombin — baseline, 6 months, 12 months Antithrombin concentration (IU) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
Plasminogen — baseline, 6 months, 12 months Plasminogen concentration (IU/ml) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
Thrombin-Antithrombin Complex — baseline, 6 months, 12 months Plasma fibrinogen concentration (ng/mL) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
Plasmin-Alpha-2-Antiplasmin Complex — 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.
Prothrombin Fragment 1+2 — 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.
Coagulation Factor X — baseline, 6 months, 12 months Coagulation Factor X concentration (IU) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
von Willebrand Factor Antigen — 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.
Plasminogen Activator Inhibitor-1 (PAI-1) — 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.
Trial sites (1)
Facility
City
Region
Status
AOU Policlinico G. Martino, UOC Cardiologia con UTIC
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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