Real life management of patients with chronic myeloid leukaemia in the United Kingdom
File(s)
Author(s)
Nesr, George Nasry Girgis
Type
Thesis
Abstract
Analysing real life data from population-based cancer registries (PBCR) informs current practice and supplements information obtained from randomised controlled clinical trials (RCTs). Furthermore, PBCR capture data about populations excluded from RCTs due to factors such as comorbidity, age and pregnancy. Chronic myeloid leukaemia (CML) is a rare haematological malignancy characterised by the BCR::ABL1 oncogene. The availability of 3+ generations of tyrosine kinase inhibitors (TKIs) and the ease of monitoring response to therapy, allowed the survival of CML patients to approach that of the normal population. “Operational cure” might be achievable for patients maintaining deep molecular responses. The UK National Registry for CML patients is a web-based PBCR, initially established as a regional registry in 2010, then expanded nationally in 2015. Data collected included baseline disease characteristics and patient demographics, and yearly follow ups for treatment and response. Maintaining the registry was hampered for various reasons, and the restart was slowed by the lack of funding and the lengthy regulatory approvals needed to accommodate changes in CML management, to include the paediatric population and pregnancy outcomes, and to link the collected data with bio banked samples to allow for collaborative research. Almost 750 CML patients from 43 centres across the UK diagnosed on or after 2009 were recruited. Median follow up duration was 4.7 years. The data provide insights into the demographics and clinical presentation of UK patients, and diagnostic and prescribing practices of UK haematologists. Despite the differences in patient characteristics between those included in the registry and those recruited to RCTs, efficacy data for TKIs was not inferior to that reported by RCTs. However, the captured monitoring practices did not conform to available guidelines. In a subgroup analysis, efficacy of generic and branded imatinib were comparable.
Version
Open Access
Date Issued
2022-09-22
Date Awarded
01/03/2023
License URL
Advisor
Apperley, Jane
Szydlo, Richard
Publisher Department
Department of Medicine
Publisher Institution
Imperial College London
Qualification Level
Doctoral
Qualification Name
Doctor of Medicine (Research) MD (Res)