Prevalence and Outcomes of Cardiac Transthyretin Amyloidosis
Research type
Research Study
Full title
Prevalence and Outcomes of Transthyretin Cardiac Amyloidosis in Cardiac Disease and Ageing
IRAS ID
253397
Contact name
James Moon
Contact email
Sponsor organisation
University College London
Clinicaltrials.gov Identifier
Z6364106/2019/09/39, UCL Data Protection registration number
Duration of Study in the UK
5 years, 0 months, 1 days
Research summary
Cardiac amyloidosis is a disease caused by the deposition of misfolded proteins in heart muscle. One of the main proteins causing cardiac amyloidosis is called transthyretin (TTR). Transthyretin amyloidosis (ATTR) can affect heart muscle, causing a cardiomyopathy (ATTR-CM). Previously thought to be a rare condition, recent studies suggest that ATTR-CM frequently coexists with other common cardiac conditions such as heart failure, heart muscle thickening, valvular heart disease and conduction abnormalities. It is likely that patients with the dual pathology of cardiac disease and ATTR-CM have higher mortality rates and benefit less from established treatment regimes. The diagnosis of ATTR-CM can be made with high sensitivity and specificity using a bone scan (99mTc-3,3-diphosphono-1,2-propanodicarboxylic acid (DPD) bone scintigraphy), avoiding the need for an invasive heart biopsy.
A recent clinical trial (ATTRACT) has shown that a TTR stabiliser, tafamidis, significantly reduces the decline of functional capacity and quality of life and reduced all-cause mortality and hospitalisation by 30% in patients with ATTR-CM. The greatest benefit was in patients with mild symptoms, emphasising the importance of early diagnosis.
This study aims to investigate the prevalence of ATTR-CM in patients with known cardiac conditions and in ageing patients without known cardiac disease. This will enable us to explore the effects of ATTR-CM on outcomes related to treatments and procedures.
This will be an observational, prospective study, recruiting patients from St Bartholomew’s Hospital and the John Radcliffe Hospital. We will perform baseline assessments, including a bone scan. Follow-up will document the clinical and cardiac features of patients and record relevant outcomes.
By establishing the prevalence and characteristics of ATTR-CM, we will be able to develop diagnostic pathways to promote its early identification. We will also be able to monitor and record the impact of treatment with both conventional therapies and emerging disease-specific treatments such as tafamidis.REC name
London - Camberwell St Giles Research Ethics Committee
REC reference
20/LO/0315
Date of REC Opinion
2 Jul 2020
REC opinion
Further Information Favourable Opinion