International sentinel site surveillance of patients with transfusional hemosiderosis treated with deferasirox in actual practice setting
| Autor: | El-Beshlawy, Amal, Inusa, Baba, Beneitez Pastor, David, Xicoy, Blanca, Duran Nieto, Maria Soledad, Bruederle, Andreas, Azmon, Amin, Gilotti, Geralyn, Elalfy, Mohsen, Universitat Autònoma de Barcelona |
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| Rok vydání: | 2018 |
| Předmět: |
Adult
Male Cancer Research medicine.medical_specialty Blood transfusion Hemosiderosis Iron Overload Adolescent medicine.medical_treatment MEDLINE 03 medical and health sciences 0302 clinical medicine Medicine Iron overload Humans Blood Transfusion Intensive care medicine Aged Retrospective Studies business.industry Deferasirox Transfusion Reaction Retrospective cohort study Hematology Iron chelation therapy Middle Aged medicine.disease Sentinel site Oncology 030220 oncology & carcinogenesis Actual practice Female Safety business 030215 immunology medicine.drug |
| Zdroj: | ResearcherID Hematology r-IGTP. Repositorio Institucional de Producción Científica del Instituto de Investigación Germans Trias i Pujol instname Dipòsit Digital de Documents de la UAB Universitat Autònoma de Barcelona |
| ISSN: | 1607-8454 |
| Popis: | Funding: This study was funded by Novartis Pharma AG 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. |
| Databáze: | OpenAIRE |
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