Quality standards for amyloidosis care
HPP supported an expert group to develop quality standards for ATTR and AL amyloidosis, translating evidence into measurable goals for health system improvement.
This project was initiated and funded by AstraZeneca.
Context
Amyloidosis is a rare, progressive and often life-threatening condition. It occurs when misfolded proteins build up in organs and tissues, such as the heart, kidneys and nerves. It can affect many areas of the body, so multidisciplinary collaboration is essential to provide comprehensive care.
But the care people receive can vary widely, depending on where they live and whether they have access to specialist amyloidosis services. Many people still wait years for a diagnosis.
Delays in recognising and investigating symptoms mean amyloidosis is often diagnosed too late, when irreversible organ damage has already occurred and the condition is harder to manage. This is leaving people vulnerable to potentially avoidable progression and early death.
Without quality standards, health systems have no common way to define good amyloidosis care, assess whether it is being achieved, or identify unwarranted variation.
What we’ve achieved
HPP facilitated a group of experts with diverse perspectives to develop a flagship set of consensus-based quality standards for ATTR and AL amyloidosis (the two most common types). We did so using a structured nominal group technique methodology – an approach that combines evidence review and expert voting and validation.
Each standard is paired with a measurable indicator, supporting health systems to benchmark services, identify gaps and improve the consistency of high-quality care delivery.
We also helped convert consensus to impact by translating the technical outputs of the project into a peer-reviewed article for Amyloid, the journal of the International Society of Amyloidosis. The article expressed support for uptake of the quality standards by professional societies, clinical networks, patient organisations and health-system decision-makers, and provides a strong foundation for implementation.
Key partners and stakeholders
This project was delivered with the support of an international expert group:
- João Agostinho, Cardiology Department, Hospital de Santa Maria, ULSSM, CAML, CCUL@RISE, Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal
- Carys Barton, British Society for Heart Failure; formerly, Cardiology Department, Imperial College Healthcare NHS Trust, London, UK
- Catarina Campos, Neurology Department, Hospital de Santa Maria, ULSSM, Lisbon, Portugal; Centro de Estudos Egas Moniz, Faculdade de Medicina da Universidade de Lisboa, Lisbon, Portugal
- Giovanni D’Alessio, L’associazione Italiana Amiloidosi fAMY Onlus, Vignola, Italy
- Thibaud Damy, Referral Center for Cardiac Amyloidosis, Réseau Amylose, F-CRIN GRACE, Clinical Epidemiology in Aging, University Hospital Henri Mondor, Créteil, France
- Keith Dares, The Canadian Amyloidosis Support Network, Toronto, Canada
- Maike F Dohrn, Department of Neurology, RWTH Aachen University Hospital, Aachen, Germany
- Nowell Fine, Cardiac Amyloidosis Clinic, University of Calgary, Calgary, Canada
- Lucia Galan, Neuromuscular Unit, Hospital Clinico San Carlos, Madrid, Spain
- David Gregory, Amyloidosis UK, London, UK
- Emmanuel Jammes, Amyloidosis Alliance, Paris, France
- Katharine McIntosh, Cardiomyopathy UK, Amersham, UK
- William Moody, Department of Cardiovascular Sciences, Queen Elizabeth Hospital, University Hospitals Birmingham NHS Foundation Trust, UK
- Eva-Britt Norberg, FAMY Norrbotten, Sweden
- Laura Obici, Amyloidosis Research and Treatment Center, Foundation IRCCS Policlinico San Matteo, Pavia, Italy
- Giovanni Palladini, Department of Molecular Medicine, University of Pavia; Amyloidosis Research and Treatment Center, Foundation IRCCS Policlinico San Matteo, Pavia, Italy
- Tomas Ripoll-Vera, Cardiology Department, Son Llatzer University Hospital; Genetic and Familial Heart Disease Unit, IdISBa, Palma De Mallorca, Spain
- Ali Yilmaz, Cardiology Department, University Hospital Münster, Münster, Germany
Project funding
This project was initiated and funded by AstraZeneca, which commissioned HPP to coordinate the work as part of a non-promotional research project. All outputs are non-promotional and evidence based. Editorial control of the peer-reviewed article was held by its authors. Expert group members were reimbursed for their time in the consensus process in line with fair market value.