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Progress in the understanding and practice of haematopoietic stem cell transplantation has
come a long way since its inception. Despite this, there is ever-increasing controversy within
most haematological malignancies about the role and timing of transplantation. Medical
practice today is governed by ‘evidence-based medicine’ and guidelines. However,
guidelines are generic, with little quality evidence base available in many scenarios to help
clinicians make decisions about transplantation in patients who fall into those controversial
categories.
The morbidity and mortality associated with allogeneic transplantation remains significant.
Autologous transplantation is a safer procedure but does not offer the same curative
potential. Consequently, decisions about which patients should be transplanted, how and
when, continue to cause controversy.
I have identified four clinical scenarios within haematopoietic stem cell transplantation where
guidelines are not clear about the specifics of practice but in which we have specific clinical
experience at St Bartholomew’s. These are the outcomes of two melphalan conditioning
doses in autologous stem cell transplantation in multiple myeloma, the outcomes of
allogeneic stem cell transplantation in multiple myeloma, the outcomes of patients with
refractory and relapsed acute myeloid leukaemia and myelodysplasia undergoing sequential
transplantation and finally the outcomes of allogeneic stem cell transplantation in lymphoma.
The aim of this thesis has been to collate and analyse patient data in each of these areas of
debate, in order to make recommendations regarding future clinical practice. Following on
from this, I have evaluated the role of haematopoietic stem cell transplantation today and its
future directions.