Retrospective analysis of outcomes of patients with β-thalassaemia
Research type
Research Study
Full title
A Retrospective Database Study of Disease Course and Clinical Outcomes in Patients with Beta Thalassemia
IRAS ID
249177
Contact name
John Porter
Contact email
Sponsor organisation
bluebird bio
Duration of Study in the UK
0 years, 10 months, 18 days
Research summary
This retrospective research study will focus on patients with beta-thalassemia who are receiving conventional treatment such as regular red blood cell transfusions and chelation therapy, and patients not regularly transfused or not transfused at all (non-transfusion dependent thalassemia, NTDT).
The aim is to systematically quantify outcomes across all beta-thalassemia patients and by sub-groups (e.g. age, gender, thalassemia sub-type (e.g. TDT vs NTDT)). The results of this study will provide context for interpretation of outcomes from other studies, as well as quantifying the disease burden present with existing treatments. The results of the study will aid future decisions about the treatment of this group of patients because it is currently not well documented.
Data currently captured at a single center will be analyzed in a retrospective fashion. For retrospective data collection patients can be living or deceased, with a documented diagnosis of beta-thalassemia. Analyses will be descriptive and comparative.
REC name
North West - Liverpool Central Research Ethics Committee
REC reference
19/NW/0313
Date of REC Opinion
3 Jun 2019
REC opinion
Favourable Opinion