Τίτλος:
A phase 3 study of deferasirox (ICL670), a once-daily oral iron
chelator, in patients with beta-thalassemia
Περίληψη:
Deferasirox (ICL670) is a once-daily oral iron chelator developed for
the treatment of chronic iron overload from blood transfusions. A
comparative phase 3 trial was conducted to demonstrate the efficacy of
deferasirox in regularly transfused patients with beta-thalassemia aged
2 years or older. Patients were randomized and received treatment with
deferasirox (n = 296) or deferoxamine (n = 290), with dosing of each
according to baseline liver iron concentration (LIC). The primary
endpoint was maintenance or reduction of LIC; secondary endpoints
included safety and tolerability, change in serum ferritin level, and
net body iron balance. In both arms, patients with LIC values of 7 mg
Fe/g dry weight (dw) or higher had significant and similar
dose-dependent reductions in LIC and serum ferritin, and effects on net
body iron balance. However, the primary endpoint was not met in the
overall population, possibly due to the fact that proportionally lower
doses of deferasirox relative to deferoxamine were administered to
patients with LIC values less than 7 mg Fe/g dw. The most common adverse
events included rash, gastrointestinal disturbances, and mild
nonprogressive increases in serum creatinine. No agranulocytosis,
arthropathy, or growth failure was associated with deferasirox
administration. Deferasirox is a promising once-daily oral therapy for
the treatment of transfusional iron overload.
Συγγραφείς:
Cappellini, MD
Cohen, A
Piga, A
Bejaoui, M
Perrone, S
and Agaoglu, L
Aydinok, Y
Kattamis, A
Kilinc, Y
Porter,
J
Capra, M
Galanello, R
Fattoum, S
Drelichman, G and
Magnano, C
Verissimo, M
Athanassiou-Metaxa, M
Giardina, P
and Kourakli-Symeonidis, A
Janka-Schaub, G
Coates, T and
Vermylen, C
Olivieri, N
Thuret, I
Opitz, H and
Ressayre-Djaffer, C
Marks, P
Alberti, D
