Improved survival of thalassaemia major in the UK and relation to T2* cardiovascular magnetic resonance.
BACKGROUND
The UK Thalassaemia Register records births, deaths and selected clinical data of patients with thalassaemia who are resident in the UK. A study of survival and causes of death was undertaken which aimed to include the possible impact of T2* cardiovascular magnetic resonance (CMR).


METHODS
The Register was updated to the end of 2003, copies of death certificates were obtained, and causes of death in beta thalassaemia major were extracted. In addition, patients who had T2* CMR assessment of cardiac iron load and/or received the oral iron chelator deferiprone were identified from clinical records.


RESULTS
The main causes of death were anaemia (before 1980), infections, complications of bone marrow transplantation and cardiac disease due to iron overload. From 1980 to 1999 there were 12.7 deaths from all causes per 1,000 patient years. Forty per cent of patients born before 1980 had T2* cardiovascular magnetic resonance between 2000 and 2003, and 36% of these patients were prescribed deferiprone before end of 2003. In 2000-2003, the death rate from all causes fell significantly to 4.3 per 1,000 patient years (-62%, p < 0.05). This was mainly driven by the reduction in the rate of deaths from iron overload which fell from 7.9 to 2.3 deaths per 1,000 patient years (-71%, p < 0.05).


CONCLUSIONS
Since 1999, there has been a marked improvement in survival in thalassaemia major in the UK, which has been mainly driven by a reduction in deaths due to cardiac iron overload. The most likely causes for this include the introduction of T2* CMR to identify myocardial siderosis and appropriate intensification of iron chelation treatment, alongside other improvements in clinical care.
Modell
Bernadette
B
UCL Centre for Health Informatics and Multiprofessional Education, Holborn Union Building, Whittington Campus, Highgate Hill, London, N19 5LW, UK. b.modell@pcps.ucl.ac.uk
Khan
Maren
M
Darlison
Matthew
M
Westwood
Mark A
MA
Ingram
David
D
Pennell
Dudley J
DJ
D000293:Adolescent; D000328:Adult; D000740:Anemia / Q000209:etiology / Q000401:mortality; D016026:Bone Marrow Transplantation / Q000009:adverse effects / Q000401:mortality; D009202:Cardiomyopathies / Q000209:etiology / Q000401:mortality* / Q000473:pathology* / Q000628:therapy; D002423:Cause of Death; D002648:Child; D002675:Child, Preschool; D003141:Communicable Diseases / Q000150:complications / Q000401:mortality; D000077543:Deferiprone; D005260:Female; D006801:Humans; D007502:Iron Chelating Agents / Q000627:therapeutic use; D019190:Iron Overload / Q000209:etiology / Q000401:mortality* / Q000473:pathology* / Q000628:therapy; D008017:Life Expectancy; D008279:Magnetic Resonance Imaging; D008297:Male; D008875:Middle Aged; D011728:Pyridones / Q000627:therapeutic use; D012042:Registries; D013997:Time Factors; D016896:Treatment Outcome; D006113:United Kingdom; D055815:Young Adult; D017086:beta-Thalassemia / Q000150:complications* / Q000401:mortality / Q000473:pathology / Q000628:therapy
D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone