Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2012 Jan 25;14(1):8.
doi: 10.1186/1532-429X-14-8.

Effects of combined deferiprone with deferoxamine on right ventricular function in thalassaemia major

Francisco Alpendurada  1 , Gill C Smith, John-Paul Carpenter, Sunil V Nair, Mark A Tanner, Winston Banya, Carlo Dessi, Renzo Galanello, John Malcolm Walker, Dudley J Pennell
Affiliations

Effects of combined deferiprone with deferoxamine on right ventricular function in thalassaemia major

Francisco Alpendurada et al. J Cardiovasc Magn Reson. .

Abstract

Background: Combination therapy with deferoxamine and oral deferiprone is superior to deferoxamine alone in removing cardiac iron and improving left ventricular ejection fraction (LVEF). The right ventricle (RV) is also affected by the toxic effects of iron and may cause additional cardiovascular perturbation. We assessed the effects of combination therapy on the RV in thalassaemia major (TM) using cardiovascular magnetic resonance (CMR).

Methods: We retrieved imaging data from 2 treatment trials and re-analyzed the data for the RV responses: Trial 1 was a randomized controlled trial (RCT) of 65 TM patients with mild-moderate cardiac siderosis receiving combination therapy or deferoxamine with placebo; Trial 2 was an open label longitudinal trial assessing combination therapy in 15 TM patients with severe iron loading.

Results: In the RCT, combination therapy with deferoxamine and deferiprone was superior to deferoxamine alone for improving RVEF (3.6 vs 0.7%, p = 0.02). The increase in RVEF was greater with lower baseline T2* 8-12 ms (4.7 vs 0.5%, p = 0.01) than with T2* 12-20 ms (2.2 vs 0.8%, p = 0.47). In patients with severe cardiac siderosis, substantial improvement in RVEF was seen with open-label combination therapy (10.5% ± 5.6%, p < 0.01).

Conclusions: In the RCT of mild to moderate cardiac iron loading, combination treatment improved RV function significantly more than deferoxamine alone. Combination treatment also improved RV function in severe cardiac siderosis. Therefore adding deferiprone to deferoxamine has beneficial effects on both RV and LV function in TM patients with cardiac siderosis.

PubMed Disclaimer

Figures

Figure 1
Figure 1
Study flow-chart.
Figure 2
Figure 2
Change in myocardial T2* (left panel) and in RVEF (right panel) over 12 months according to treatment arm. Vertical lines represent standard error.
Figure 3
Figure 3
Change in RVEF over 12 months according to treatment arm and myocardial T2* at baseline (T2* 8-12 ms on left panel, T2* 12-20 ms on right panel). Vertical lines represent standard error.
Figure 4
Figure 4
Breakdown of improvement in RVEF (%) for different groups according to chelation therapy and myocardial T2* baseline. Vertical lines represent standard error.
Figure 5
Figure 5
Correlation of change in RVEF with change in LVEF over 12 months.
See this image and copyright information in PMC

References

    1. Zurlo MG, De Stefano P, Borgna-Pignatti C, Di Palma A, Piga A, Melevendi C, Di Gregorio F, Burattini MG, Terzoli S. Survival and causes of death in thalassaemia major. Lancet. 1989;8653:27–30. - PubMed
    1. Olivieri NF, Nathan DG, MacMillan JH, Wayne AS, Liu PP, McGee A, Martin M, Koren G, Cohen AR. Survival in medically treated patients with homozygous beta-thalassemia. N Engl J Med. 1994;331:574–8. doi: 10.1056/NEJM199409013310903. - DOI - PubMed
    1. Borgna-Pignatti C, Rugolotto S, De Stefano P, Zhao H, Cappellini MD, Del Vecchio GC, Romeo MA, Forni GL, Gamberini MR, Ghilardi R, Piga A, Cnaan A. Survival and complications in patients with thalassemia major treated with transfusion and deferoxamine. Haematologica. 2004;89:1187–193. - PubMed
    1. Anderson LJ, Wonke B, Prescott E, Holden S, Walker JM, Pennell DJ. Comparison of effects of oral deferiprone and subcutaneous desferrioxamine on myocardial iron levels and ventricular function in beta thalassemia. Lancet. 2002;360:516–20. doi: 10.1016/S0140-6736(02)09740-4. - DOI - PubMed
    1. Modell B, Khan M, Darlison M. Survival in beta-thalassaemia major in the UK: data from the UK Thalassaemia Register. Lancet. 2000;355:2051–2. doi: 10.1016/S0140-6736(00)02357-6. - DOI - PubMed

Publication types

MeSH terms