Sachchithanantham, Sajitha; (2017) A profile of AL amyloidosis with rare sub-types, novel investigations and prognostic markers. Doctoral thesis (M.D(Res)), UCL (University College London).

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Abstract

Background Systemic AL amyloidosis is a rare complication of immunoglobulin light chain secreting B cell clonal disorders. Much progress has been made in the recent years in the management of AL amyloidosis. Yet, certain patient groups continue to fare badly, posing a challenge to the treating physicians. Aims To describe the clinical features and outcomes of the challenging subgroups of patients with AL amyloidosis such as elderly patients and those with rare subtypes – IgD and IgM related amyloidosis. To explore the role of 99mTc-DPD scintigraphy in imaging soft tissue AL amyloid deposits and look at possible risk stratifying methods based on plasma cell phenotype and serum clonal markers at presentation. To evaluate the effectiveness of the novel agent, bortezomib as front line therapy in AL amyloidosis. Results and Conclusion Treatment of systemic AL amyloidosis in the elderly is challenging, yet, treatment of carefully selected older patients with novel therapies with low toxicity profile, results in improved survival. The clinical profile of IgD amyloidosis is similar to that of AL in general but the long term outcome appears poor. In contrast, IgM related amyloidosis has some distinct features and the underlying B cell clone needs to be accurately characterised to direct the choice of therapy. The adverse outcome in this latter group appears to be associated with cardiac, liver and nerve involvement. The role of 99mTc-DPD scintigraphy in imaging soft tissue AL amyloidosis is promising and requires further studies. Multicolour flow cytometry and heavy light chain measurement seem valuable in assessing the impact of plasma cell clones and degree of immunosuppression on prognosis respectively. Bortezomib based treatment is effective in achieving deep clonal response in patients without cardiac amyloidosis and those with early disease. Those with advanced cardiac involvement continue to pose a challenge and are in need of more effective therapies.

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