Cardiac disease is the cause of approximately 70 percent
of deaths in beta thalassaemia major. Cardiovascular magnetic resonance
(CMR) can measure cardiac iron deposition through the magnetic relaxation
parameter T2*. This allows the determination of best chelation strategies
for the heart by direct assessment of the clearance of myocardial iron.
Deferoxamine (constant intravenous) was assessed in seven patients presenting
with heart failure. Of the six survivors, cardiac T2* improved significantly
over one year, but clearance was very slow. A case-controlled study comparing
deferiprone with deferoxamine showed lower myocardial iron and superior
ejection fraction in patients on deferiprone. Two prospective, randomized,
controlled, blinded trials have been conducted to explore this result further.
The first trial compared deferiprone 100mg with deferoxamine and showed
superior efficacy for deferiprone in removing cardiac iron. The second trial
compared combination therapy vs. deferoxamine alone, and is now being analyzed.
Direct measures of myocardial iron using myocardial T2* are giving new insights
into cardiac deaths in thalassaemia. We are working with TIF and NIH grants
to validate other centres worldwide and to calibrate the method. So far,
oral chelation with deferiprone appears to be more effective than deferoxamine
in removing cardiac iron. |