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Clinical Trial
. 2012 Jun;97(6):842-8.
doi: 10.3324/haematol.2011.049957. Epub 2012 Jan 22.

Deferasirox for up to 3 years leads to continued improvement of myocardial T2* in patients with β-thalassemia major

Dudley J Pennell  1 , John B Porter, Maria Domenica Cappellini, Lee Lee Chan, Amal El-Beshlawy, Yesim Aydinok, Hishamshah Ibrahim, Chi-Kong Li, Vip Viprakasit, Mohsen S Elalfy, Antonis Kattamis, Gillian Smith, Dany Habr, Gabor Domokos, Bernard Roubert, Ali Taher
Affiliations
Clinical Trial

Deferasirox for up to 3 years leads to continued improvement of myocardial T2* in patients with β-thalassemia major

Dudley J Pennell et al. Haematologica. 2012 Jun.

Abstract

Background: Prospective data on cardiac iron removal are limited beyond one year and longer-term studies are, therefore, important.

Design and methods: Seventy-one patients in the EPIC cardiac substudy elected to continue into the 3(rd) year, allowing cardiac iron removal to be analyzed over three years.

Results: Mean deferasirox dose during year 3 was 33.6 ± 9.8 mg/kg per day. Myocardial T2*, assessed by cardiovascular magnetic resonance, significantly increased from 12.0 ms ± 39.1% at baseline to 17.1 ms ± 62.0% at end of study (P<0.001), corresponding to a decrease in cardiac iron concentration (based on ad hoc analysis of T2*) from 2.43 ± 1.2 mg Fe/g dry weight (dw) at baseline to 1.80 ± 1.4 mg Fe/g dw at end of study (P<0.001). After three years, 68.1% of patients with baseline T2* 10 to <20 ms normalized (≥ 20 ms) and 50.0% of patients with baseline T2* >5 to <10 ms improved to 10 to <20 ms. There was no significant variation in left ventricular ejection fraction over the three years. No deaths occurred and the most common investigator-assessed drug-related adverse event in year 3 was increased serum creatinine (n = 9, 12.7%).

Conclusions: Three years of deferasirox treatment along with a clinically manageable safety profile significantly reduced cardiac iron overload versus baseline and normalized T2* in 68.1% (32 of 47) of patients with T2* 10 to <20 ms.

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Figures

Figure 1.
Figure 1.
(A) Geometric mean cardiac T2* ± 95% CI during deferasirox treatment for three years; and (B) shift in proportion of patients with severe, mild-to-moderate and normalized cardiac T2* values at baseline and EOS.
Figure 2.
Figure 2.
Mean cardiac iron concentration ± SD during 3-year deferasirox treatment.
Figure 3.
Figure 3.
Mean LVEF ± SD during 3-year deferasirox treatment.
Figure 4.
Figure 4.
(A) Mean LIC ± SD; and (B) median serum ferritin ± interquartile range during deferasirox treatment for up to three years.
Figure 5.
Figure 5.
(A) Frequency of the most common (≥5%) investigator-assessed drug-related AEs; (B) mean creatinine clearance ± SD over time during deferasirox treatment for up to three years (Cockcroft–Gault formula); (C) mean creatinine clearance ± SD over time during deferasirox treatment for up to three years (MDRD formula).
See this image and copyright information in PMC

References

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