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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. Departament d'Hematologia)
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, Cairo)
Universitat Autònoma de Barcelona

Date: 2019
Abstract: 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.
Note: Funding: This study was funded by Novartis Pharma AG
Rights: 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. Creative Commons
Language: Anglès
Document: article ; recerca ; publishedVersion
Subject: Deferasirox ; Iron overload ; Iron chelation therapy ; Safety
Published in: Hematology, 2019 , ISSN 1607-8454

DOI: 10.1080/16078454.2018.1558758

10 p, 1.6 MB

The record appears in these collections:
Research literature > UAB research groups literature > Research Centres and Groups (scientific output) > Health sciences and biosciences > Institut d'Investigació en Ciencies de la Salut Germans Trias i Pujol (IGTP)
Articles > Research articles
Articles > Published articles

 Record created 2019-04-02, last modified 2020-08-11

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