Objective: To estimate the burden of transthyretin cardiac amyloidosis (ATTR-CA) through a cross- sectional 'snapshot' of Australian Amyloidosis Network (AAN) and New Zealand (NZ) specialist amyloidosis clinics.
Design, Setting & Participants: A prospective survey was performed of seven AAN/ specialist amyloidosis clinics across Australia and NZ. All centres were invited to contribute data; participating centres provided clinical and demographic data for patients with ATTR-CA reviewed in the 2022 calendar year.
In newly diagnosed transplant-ineligible patients with myeloma, daratumumab has improved outcomes when added to the standard-of-care regimens. In a randomized trial, we tested whether similar improvements would be observed when daratumumab was added to the bortezomib, cyclophosphamide, and dexamethasone (VCD) regimen. Transplant-ineligible patients with untreated myeloma were randomized to receive VCD or VCD plus daratumumab (VCDD).
View Article and Find Full Text PDFOver the past 5 years, early diagnosis of and new treatments for cardiac amyloidosis (CA) have emerged that hold promise for early intervention. These include non-invasive diagnostic tests and disease modifying therapies. Recently, CA has been one of the first types of cardiomyopathy to be treated with gene editing techniques.
View Article and Find Full Text PDFObjectives: The treatment of hereditary transthyretin amyloidosis polyneuropathy (ATTRv-PN) has been revolutionised by genetic therapies, with dramatic improvements in patient outcomes. Whilst the optimal timing of treatment initiation remains unknown, early treatment is desirable. Consequently, the aim of the study was to develop biomarkers of early nerve dysfunction in ATTRv-PN.
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