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Title: Cardiac amyloidosis : clinical characteristics, prognostic factors and treatment
Author: Patel, Ketna
ISNI:       0000 0004 9352 4478
Awarding Body: UCL (University College London)
Current Institution: University College London (University of London)
Date of Award: 2020
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Cardiac amyloidosis occurs when misfolded protein fibrils are deposited in the extracellular space of the myocardium. The most common causes for cardiac amyloidosis are light chain (AL) amyloidosis and transthyretin (ATTR) amyloidosis. ATTR amyloidosis can occur with ageing due to the accumulation of wild-type transthyretin or can due to an inherited genetic mutation. One of the more common inherited forms in the United Kingdom (UK) is associated with the V122I mutation. I have described the clinical features, electrocardiogram and multimodality imaging findings in the most common forms of ATTR cardiac amyloidosis diagnosed in the UK. I found significant differences between ATTR amyloidosis types. In particular, those with V122I associated ATTR cardiac amyloidosis present at a younger age than those with wild-type disease but have a more advanced cardiac phenotype at diagnosis and a worse survival. I identified predictors of survival in transthyretin cardiac amyloidosis which were predominantly those associated with a more severe cardiac phenotype and found that the most common form of death in these patients is from heart failure. I examined the use of DPD in ATTR and AL cardiac amyloidosis and showed that higher DPD grades are associated with more severe cardiac disease in ATTR amyloidosis. However, DPD grade does not predict survival in ATTR amyloidosis and I demonstrated that this is also the case in AL amyloidosis. I conducted a retrospective study to examine the effect of the non-steroidal anti-inflammatory drug, diflunisal, in ATTR amyloidosis. I found that even in highly selected patients, diflunisal was poorly tolerated and I did not find any evidence for benefit. I described the UK experience of a phase 2 trial of a silencing RNA treatment, revusiran. I described challenges to recruitment and patient characteristics for those I recruited.
Supervisor: Not available Sponsor: Not available
Qualification Name: Thesis (Ph.D.) Qualification Level: Doctoral
EThOS ID:  DOI: Not available