📋 Tender Overview
Project Type
Coordinated Research Project
Project Code
E13055
CRP
2317
Approved Date
9 September 2021
Status
Active - Ongoing
Start Date
19 October 2021
Expected End Date
31 December 2025
Participating Countries
ArgentinaAustraliaBrazilCroatiaCubaGreeceIndiaItalyLatviaMalaysiaMexicoSerbiaSouth AfricaSpainSwedenTürkiyeUnited States of AmericaUruguay
Description
Heart failure (HF) with preserved ejection fraction (HFpEF) is a major public health problem with no effective therapy until recently, and substantial underdiagnosis. One of the deadliest forms of HFpEF, estimated to affect 13-18% of adults >65 years with HFpEF, is transthyretin amyloid cardiomyopathy (ATTR-CMP) with a median survival of 25-41 months. Fortunately, recent therapeutic breakthrough— tafamidis— has improved survival, alleviated symptoms, and reduced HF hospitalizations from ATTR-CMP. However, we are unable to effectively harness the recent therapeutic breakthroughs to treat this important cause of HFpEF due to a key barrier—underdiagnosis of ATTR-CMP. The two key factors contributing to underdiagnosis as well as a delay in diagnosis of cardiac amyloidosis are (1) a lack of knowledge about amyloidosis due to its perceived rarity and (2) a lack of expertise in Tc-99m-PYP imaging. Tc-99m-PYP SPECT is a simple nuclear cardiology test that can accurately diagnose ATTR-CMP and has virtually eliminated the need for endomyocardial biopsy. Although access to Tc-99m-PYP SPECT and technical expertise exists, the practical application of PYP imaging to diagnose ATTR-CMP remains limited in many countries. Moreover, the current multisocietal diagnostic criteria were developed by physicians with amyloidosis expertise from Europe and America; whether these same criteria are valid in an ethnically and/or socio-economic diverse cohort of individuals in the rest of world, is not known. We expect this project to build expertise on high-quality Tc-99m-PYP SPECT imaging. The goal of this project is to develop sustainable global expertise to perform high quality PYP imaging to accurately diagnose ATTR-CMP.
Rationale of the coordinated research project
Despite availability of non-invasive methods, timely diagnosis of ATTR-CMP remains challenging. The challenge of accurate and timely diagnosis of ATTR-CMP was recently highlighted in a patient-focused survey conducted by the Amyloidosis Research Consortium (ARC).33 Before a correct diagnosis was made, 57% of hereditary (h)ATTR and 39% of wild type (wt)ATTR patients received a misdiagnosis. Of those patients who were misdiagnosed, a majority (76%, wtATTR; 75%, hATTR) received treatment for the misdiagnosed condition. 43% and 26% of patients reported being treated with beta blockers and ACE inhibitors, respectively; both of these medications are poorly tolerated in ATTR and may result in worse outcomes. 44% of patients saw =3 physicians before receiving the correct diagnosis, and 17% saw =5, suggesting missed opportunities for diagnosi
