International sentinel site surveillance of patients with transfusional hemosiderosis treated with deferasirox in actual practice setting
El-Beshlawy, Amal (Cairo University)
Inusa, Baba (Evelina Children's Hospital)
Beneitez Pastor, David (Hospital Universitari Vall d'Hebron)
Xicoy, Blanca (Institut Germans Trias i Pujol. Institut de Recerca contra la Leucèmia Josep Carreras)
Duran Nieto, Maria Soledad (Hospital Materno Infantil de Jaén)
Bruederle, Andreas (Novartis Pharma AG)
Azmon, Amin (Novartis Pharma AG, Switzerland)
Gilotti, Geralyn (Novartis Pharmaceuticals Corporation, USA)
Elalfy, Mohsen (Ain Shams University (El Caire, Egipte))
Universitat Autònoma de Barcelona

Data:	2019
Resum:	Objective:The study evaluates the long-term deferasirox treatment of adult and pediatricpatients with chronic transfusional iron overload in clinical practice. Methods:In this non-interventional study, patients were observed for up to 3 years frominitiation of deferasirox treatment both prospectively and retrospectively for up to 1 yearprior to enrollment. The primary end points were the proportion of patients with≥1 notableincrease in serum creatinine (SCr), and≥1 notable increase in alanine aminotransferase (ALT). Results:Overall, 120 patients were enrolled and 51 completed the study, with a limited 3-yeardropout rate of 12. 5% due to adverse events (AEs). Increase in SCr > 33% above baseline and theage-adjusted ULN (upper limit of normal) was observed in 14 patients (95%CI, 7. 1-19. 2). The ALTlevels >5 × ULN was observed in 1 patient. Most frequent AEs reported during treatment withdeferasirox include gastrointestinal disturbances. Conclusions:The long-term treatment with deferasirox was manageable in most transfusion-dependent patients with no unexpected safetyfindings. Regular monitoring and an adjusteddeferasirox dosing strategy per local labels allowed continued iron chelation treatment andcontrol of transfusional iron in the majority of patients on study.
Nota:	Funding: This study was funded by Novartis Pharma AG
Drets:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original.
Llengua:	Anglès
Document:	Article ; recerca ; Versió publicada
Matèria:	Deferasirox ; Iron overload ; Iron chelation therapy ; Safety
Publicat a:	Hematology, 2019 , ISSN 1607-8454

DOI: 10.1080/16078454.2018.1558758

10 p, 1.6 MB

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Documents de recerca > Documents dels grups de recerca de la UAB > Centres i grups de recerca (producció científica) > Ciències de la salut i biociències > Institut d'Investigació en Ciencies de la Salut Germans Trias i Pujol (IGTP) > Institut de Recerca contra la Leucèmia Josep Carreras
Articles > Articles de recerca
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