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Background: Hereditary transthyretin-mediated amyloidosis is a rare, progressive and potentially life-limiting multisystem disease, affecting every aspect of a patient's life., Objectives: This online international Delphi survey aimed to evolve clinical-patient-led practical guidance, to inspire and encourage a holistic approach to care that is managed in specialist settings by multidisciplinary teams and supported by allied healthcare professionals (HCPs) and patient advocacy groups (PAGs)., Design: A 14-member joint patient advocate-HCP primary panel was convened including representation from PAGs and key clinical specialties (neurology, cardiology, internal medicine, physiotherapy, clinical psychology, dietetics and specialist nursing). Guidance evolved on the care provision needed to support seven core goals: early diagnosis and treatment; disease monitoring and organisation of care; maintenance of physical and mental health; family-centred care and caregiver support; patient-doctor dialogue; access to social support and social networking., Participants: From June to October 2022, 252 HCPs and 51 PAG representatives from 27 countries were invited to participate in a Delphi survey. Of the 122 respondents who answered at least one survey question, most were HCPs (100, 82%) from specialist centres; the remainder were PAG representatives (22, 18%)., Main Outcome Measure: Both level of agreement and feasibility in practice of each recommendation was tested by two anonymised online Delphi voting rounds., Results: Based on an a priori threshold for consensus of ≥75% agreement, the clinical-patient community endorsed all but one recommendation. However, only 17/49 (35%) recommendations were identified by most HCPs as a core part of routine care; the remainder (32/49 (65%)) were identified as part of core care by <50% of HCPs respondents, or as largely achievable by 30%-45% of HCPs. By comparison, PAGs recorded lower implementation levels., Conclusions: Further consideration is needed on how to evolve multidisciplinary services (supported by allied HCPs and PAGs) to address the complex needs of those affected by this disease., Competing Interests: Competing interests: LO has received speaker honoraria from Pfizer, Akcea and Alnylam; and has received honoraria for participation on advisory panels from Alnylam, Sobi, AstraZeneca, Novo Nordisk, and Pfizer. RC has received honoraria for participation in webinars from Alnylam; has received honoraria for participation in symposia from Alnylam; and has received honoraria for participation on an advisory panel from Alnylam. JA has received honoraria for participation on an advisory panel from Alnylam. CB has received honoraria for participating in symposia from Alnylam; and has received honoraria for participation on an advisory panel from Alnylam. TB received honoraria for participation in webinars from Alnylam; and has received honoraria for participation on an advisory panel from Alnylam. IC has received research funding from Pfizer; has received honoraria for participation in advisory boards from Pfizer, Alnylam, and Akcea; and has received financial support as primary investigator from Alnylam, and Ionis. AF has received honoraria for participation on an advisory panel from Alnylam. FK has received honoraria for participation in educational presentations from Alnylam; and has received honoraria for participation on an advisory panel from Alnylam. TL is an employee and stockholder of Alnylam Pharmaceuticals. L-OL received honoraria for participation on an advisory panel from Alnylam. AM has received honoraria for participation in webinars from Alnylam; and has received honoraria for participation on an advisory panel from Alnylam. VN has received honoraria for participation on an advisory panel from Alnylam. TC has received speaker honoraria from Pfizer, Alnylam, GlaxoSmithKline, Prothena and Akcea; has received expenses support for scientific meetings from Pfizer, Alnylam, Ionis, and Biogen; and has received honoraria for participation on an advisory panel from Alnylam. FD has no competing interests to declare., (© Author(s) (or their employer(s)) 2023. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Transthyretin cardiac amyloidosis (ATTR-CA) is a systemic disorder resulting from the extracellular deposition of amyloid fibrils of misfolded transthyretin protein in the heart. ATTR-CA is a life-threatening disease, which can be caused by progressive deposition of wild type transthyretin (wtATTR) or by aggregation of an inherited mutated variant of transthyretin (mATTR). mATTR Is a rare condition transmitted in an autosomal dominant manner with incomplete penetrance, causing heterogenous phenotypes which can range from predominant neuropathic involvement, predominant cardiomyopathy, or mixed. Diagnosis of ATTR-CA is complex and requires integration of different imaging tools (echocardiography, bone scintigraphy, magnetic resonance) with genetics, clinical signs, laboratory tests, and histology. In recent years, new therapeutic agents have shown good efficacy and impact on survival and quality of life in this subset of patients, nevertheless patients affected by ATTR-CA may still carry an unfavorable prognosis, thus highlighting the need for new therapies. This review aims to assess cardiovascular involvement, diagnosis, and management of patients affected by ATTR-CA., Competing Interests: Disclosure The authors have nothing to disclose., (Copyright © 2021 Elsevier Inc. All rights reserved.)