@article{3147993,
    title = "Deferasirox effectively decreases iron burden in patients with double
heterozygous HbS/beta-thalassemia",
    author = "Voskaridou, Ersi and Plata, Eleni and Douskou, Marousa and Sioni, and Anastasia and Mpoutou, Efrosini and Christoulas, Dimitrios and and Dimopoulou, Maria and Terpos, Evangelos",
    journal = "Annals of Hematology",
    year = "2011",
    volume = "90",
    number = "1",
    pages = "11-15",
    publisher = "Springer-Verlag",
    issn = "0939-5555, 1432-0584",
    doi = "10.1007/s00277-010-1029-7",
    keywords = "Double heterozygous HbS/beta-thalassemia; Sickle cell syndromes;
Deferasirox; Iron overload; Liver; Heart",
    abstract = "Iron overload is present in several cases of double heterozygous
sickle-cell/beta-thalassemia (HbS/beta-thal). Deferasirox is an orally
administered iron chelator which is effective on iron overloaded
patients with transfusion-dependent anemia. The aim of this study was to
investigate the efficacy and safety of deferasirox on HbS/beta-thal
patients with iron overload. We evaluated 31 adult patients with
HbS/beta-thal (14M/17F; median age 41 years) who had serum ferritin
levels > 1,000 ng/mL and who were sporadically transfused. Total iron
burden was monitored by measuring serum ferritin levels before and
monthly after starting deferasirox, while liver iron concentration and
cardiac iron burden were measured by magnetic resonance imaging (MRI) T2
and T2* parameters at baseline and 12 months after deferasirox
treatment. Deferasirox managed to reduce the mean serum ferritin levels
after 12 months of treatment from 1,989 +/- 923 to 1,008 +/- 776 ng/mL
(P < 0.001). This reduction was accompanied by a significant improvement
on MRI T2* of the liver (from 3.9 +/- 3.2 to 5.8 +/- 3.1 ms; P < 0.01)
and by a comparable improvement of biochemical parameters of liver
function. Mild nausea and diarrhea of grade 1/2 were reported in 25% of
patients within the first month of treatment, but did not re-occur.
These data indicate that deferasirox provided effective control of iron
levels (mainly of the liver) in minimally transfused patients with
HbS/beta-thal, without significant adverse events, at similar doses to
those studied widely for the treatment of patients with thalassemia
syndromes."
}