Sharpley, Faye Amelia;
(2023)
Novel approaches in the prognostication, monitoring and management of light chain amyloidosis.
Doctoral thesis (M.D(Res)), UCL (University College London).
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Abstract
Background: Systemic light chain (AL) amyloidosis is a rare disorder characterised by the production of abnormal clonal light chains, which mis-fold and deposit as amyloid fibrils within the tissues, with progressive organ dysfunction. Significant progress has been made in the field of AL amyloidosis, but improvements can still be made in the stratification of patients, the monitoring of patients who achieve a clonal remission and in the treatment of patients, both with autologous stem cell transplantation (ASCT) and at relapse. / Aims: • To explore the merits of mass spectrometry as a novel diagnostic technique. • To analyse the features of Mayo stage 1 patients to help identify variables predictive for survival. • To assess the outcome of patients treated with an autologous stem cell transplant and to compare outcomes with patients treated with standard first line chemotherapy. • To analyse the outcomes of relapsed patients treated with the immunomodulatory drug pomalidomide. • To explore the complications of treatment including reactivation of cytomegalovirus infection. • To assess if amyloidosis can complicate solid organ transplantation. / Results and Conclusions: • Mass spectrometry can accurately identify and quantify a monoclonal light chain component, even in patients in a complete light chain response by current serological methods. • The cardiac biomarker N- terminal B-natriuretic peptide still has a prognostic value in Mayo stage 1 patients. • ASCT remains a safe and effective treatment with outcomes comparable to the large American transplant centres, but with no survival benefit over standard first line treatment. • Pomalidomide is a therapeutic option for multiply relapsed AL amyloidosis patients but responses are not as sustained nor as deep in the real-world setting. • There is a substantial risk of cytomegalovirus reactivation in patients treated with bortezomib • Solid organ transplantation appears to be a genuine risk factor for the development of both AL and AA amyloidosis.
| Type: | Thesis (Doctoral) |
|---|---|
| Qualification: | M.D(Res) |
| Title: | Novel approaches in the prognostication, monitoring and management of light chain amyloidosis |
| Open access status: | An open access version is available from UCL Discovery |
| Language: | English |
| Additional information: | Copyright © The Author 2023. Original content in this thesis is licensed under the terms of the Creative Commons Attribution-NonCommercial 4.0 International (CC BY-NC 4.0) Licence (https://creativecommons.org/licenses/by-nc/4.0/). Any third-party copyright material present remains the property of its respective owner(s) and is licensed under its existing terms. Access may initially be restricted at the author’s request. |
| UCL classification: | UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine > Inflammation UCL |
| URI: | https://discovery-pp.ucl.ac.uk/id/eprint/10180212 |
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