RT Journal Article
T1 International sentinel site surveillance of patients with transfusional hemosiderosis treated with deferasirox in actual practice setting.
A1 El-Beshlawy, Amal
A1 Inusa, Baba
A1 Beneitez Pastor, David
A1 Xicoy, Blanca
A1 Soledada Duran Nieto, Maria
A1 Bruederle, Andreas
A1 Azmon, Amin
A1 Gilotti, Geralyn
A1 Elalfy, Mohsen
K1 Deferasirox
K1 iron chelation therapy
K1 iron overload
K1 safety
AB The study evaluates the long-term deferasirox treatment of adult and pediatric patients with chronic transfusional iron overload in clinical practice. In this non-interventional study, patients were observed for up to 3 years from initiation of deferasirox treatment both prospectively and retrospectively for up to 1 year prior to enrollment. The primary end points were the proportion of patients with ≥1 notable increase in serum creatinine (SCr), and ≥1 notable increase in alanine aminotransferase (ALT). Overall, 120 patients were enrolled and 51 completed the study, with a limited 3-year dropout rate of 12.5% due to adverse events (AEs). Increase in SCr > 33% above baseline and the age-adjusted ULN (upper limit of normal) was observed in 14 patients (95%CI, 7.1-19.2). The ALT levels >5 × ULN was observed in 1 patient. Most frequent AEs reported during treatment with deferasirox include gastrointestinal disturbances. The long-term treatment with deferasirox was manageable in most transfusion-dependent patients with no unexpected safety findings. Regular monitoring and an adjusted deferasirox dosing strategy per local labels allowed continued iron chelation treatment and control of transfusional iron in the majority of patients on study.
YR 2019
FD 2019
LK http://hdl.handle.net/10668/13318
UL http://hdl.handle.net/10668/13318
LA en
DS RISalud
RD Apr 10, 2025