Publication: International sentinel site surveillance of patients with transfusional hemosiderosis treated with deferasirox in actual practice setting.
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Date
2019
Authors
El-Beshlawy, Amal
Inusa, Baba
Beneitez Pastor, David
Xicoy, Blanca
Soledada Duran Nieto, Maria
Bruederle, Andreas
Azmon, Amin
Gilotti, Geralyn
Elalfy, Mohsen
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Abstract
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.
Description
MeSH Terms
Adolescent
Adult
Aged
Blood Transfusion
Deferasirox
Female
Hemosiderosis
Humans
Iron Overload
Male
Middle Aged
Retrospective Studies
Transfusion Reaction
Adult
Aged
Blood Transfusion
Deferasirox
Female
Hemosiderosis
Humans
Iron Overload
Male
Middle Aged
Retrospective Studies
Transfusion Reaction
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Keywords
Deferasirox, iron chelation therapy, iron overload, safety