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|Title: ||Feasibility and utility of a sickle cell disease registry for research and patient management|
|Authors: ||Gilmore, Annette|
|Advisors: ||Farrow, A|
|Keywords: ||Clinical databases|
|Publication Date: ||2009|
|Publisher: ||Brunel University School of Health Sciences and Social Care PhD Theses|
|Abstract: ||This thesis aimed to evaluate the feasibility and utility of a sickle cell disease registry for
clinical patient management and research. Five hospitals out of nine in the North West
London health region participated in the registry, with 78 percent coverage of the sickle
cell disease population. There was 80% case ascertainment in participating hospitals.
Aggregated anonymised demographic and diagnostic data was collected for all
haemoglobinopathy patients. This provided the core dataset for quantifying prevalence of
sickle cell and thalassaemia and mapping local hospital workloads and service
requirements. Thirteen percent of HbSS adult patients were taking hydroxycarbamide.
The cohort of patients treated with hydroxycarbamide was evaluated. Sixty two of the 80
patients started on treatment were included. Follow-up was censored after 9 years, totalling
249 person-years of data with a median follow-up of three years (IQR, 1-6). Results
showed that haematological benefits were maintained in the long-term with treatment, but
evidence of long-term clinical effectiveness was less strong. This appeared to be due to the
patterns of clinical management in everyday practice. Patients tend to be treated with
modest doses of hydroxycarbamide due to intolerance or inability to attain or maintain
maximum tolerated dose. For example maximum tolerated dose was the aim of treatment
for 91% of patients but it was achieved for 65% of participants. Non- compliance with
treatment and monitoring schedule was the main reason for non- attainment.
Results suggest that it is sensible to strive for maximum tolerated dose to ensure therapy
remains effective, but with more realistic expectations of the dose patients can attain and
maintain. Doses in adult patients average 20mg/kg/day and 25mg/kg/day in children. Adult
patients may be able to achieve a higher dose, if there was more stringent monitoring and
improved management of non-compliance.
The North West London HU Sub-Registry proved useful for measuring long-term
effectiveness and tolerability of hydroxycarbamide. Routinely collected data was utilized
for both clinical management and research purposes. The novelty lay in examination of the
nuances of routine clinical practice. An electronic patient record was developed as a
clinical management tool. It is the first study reporting long-term outcomes for UK sickle
cell disease patients on hydroxycarbamide.
Findings should help clinicians devise effective treatment protocols and strategies for
managing patients commenced on this therapy. Interventions need to be targeted at
increasing utilisation, patient adherence and persistence with treatment. The electronic
patient record could be used to maximise treatment benefit and improve adherence. More
effective involvement of the multidisciplinary team and primary care colleagues in patient
education and management should improve usage. Patients and carers need up to date and
easy to assimilate information to make informed decisions about treatment options.
Maintaining a SCD registry is challenging. Models which operate as clinical information
systems provide an incentive for participation. These enable active involvement of local
care providers in registry management and the ability to keep and utilize their own data.
Clinicians require accurate and current data for patient management and to enable them to
benchmark their local outcomes against national outcomes and care standards.|
|Description: ||This thesis was submitted for the degree of Doctor of Philosophy and awarded by Brunel University.|
|Appears in Collections:||Health|
School of Health Sciences and Social Care Theses
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