Study to evaluate the management of patients with β-thalassaemia
Research type
Research Study
Full title
An observational study to evaluate the routine management, healthcare resource use and outcomes for patients with transfusion-dependent β-thalassaemia treated in the United Kingdom
IRAS ID
238480
Contact name
Kate Ryan
Contact email
Sponsor organisation
bluebird bio
Duration of Study in the UK
0 years, 4 months, 27 days
Research summary
This study will describe current real world routine management for patients with transfusion-dependent thalassaemia (TDT) in the UK. The results of the study will aid future decisions about the treatment of this group of patients because it is currently not well documented.
Multi-centre, observational mixed-methodology study involves a retrospective chart review and cross-sectional survey of patients with TDT and their parents/carers. Conducted in seven to nine NHS secondary/tertiary centres in the UK. Approximately 200 patients will take part; 50 patients aged under 12 years and 150 patients aged 12 or more years.
For retrospective data collection patients can be living or deceased, with a documented diagnosis of TDT and date of first transfusion at least two years prior to date of data collection. Ineligible patients are those who have less than 2 years continuous data available; had a stem cell transplant and no disease recurrence requiring transfusions; no written informed consent (if living) or were in a clinical trial.
Retrospective data will be collected from patients’ medical records by trained data collectors with a duty of confidentiality. All living patients will be invited to consent and allow researchers access to their medical records for the purpose of data collection. Since no consent can be obtained where the patient is deceased and contacting relatives of deceased patients is likely to cause distress, members of the direct care team will collect data from identifiable records and provide pseudo-anonymised data to researchers for analysis.
For the cross-sectional survey, participants (consenting living patients and parent/carers) will complete questionnaires, at one time point only, relating to their experiences of thalassaemia from the last seven days.
Data collection will be over a 5 month period. Analyses will be descriptive and comparative.
REC name
London - Harrow Research Ethics Committee
REC reference
17/LO/2114
Date of REC Opinion
14 Mar 2018
REC opinion
Further Information Favourable Opinion