Article
Adverse drug reactions of the iron chelator deferiprone during mono- and simultaneous combination therapy in Chinese paediatric thalassemia patients
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Authors
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Sebastian Botzenhardt
- Department of Paediatrics and Adolescent Medicine, University Hospital Erlangen, Erlangen, Germany
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Chor W. Sing
- Department of Pharmacology and Pharmacy, Li Ka Shing Faculty of Medicine, University of Hong Kong, Hong Kong, China
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Ian C. K. Wong
- Department of Pharmacology and Pharmacy, Li Ka Shing Faculty of Medicine, University of Hong Kong, Hong Kong, China
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Godfrey C. F. Chan
- Department of Paediatrics and Adolescent Medicine, Paediatric Haematology, Li Ka Shing Faculty of Medicine, University of Hong Kong, Hong Kong, China
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Lisa Y. L. Wong
- Department of Pharmacology and Pharmacy, Li Ka Shing Faculty of Medicine, University of Hong Kong, Hong Kong, China
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Mariagrazia Felisi
- Consorzio per Valutazioni Biologiche e Farmacologiche, Pavia, Italy
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Wolfgang Rascher
- Department of Paediatrics and Adolescent Medicine, University Hospital Erlangen, Erlangen, Germany
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Adriana Ceci
- Consorzio per Valutazioni Biologiche e Farmacologiche, Pavia, Italy
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Antje Neubert
- Department of Paediatrics and Adolescent Medicine, University Hospital Erlangen, Erlangen, Germany
| Published: | November 18, 2014 |
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Outline
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Background: There is a lack of knowledge regarding the incidence of adverse drug reactions (ADR) to the iron chelator deferiprone alone or in simultaneous combination with deferoxamine in transfusion-dependent paediatric thalassemia patients. Particularly the incidence rates of neutropenia and agranulocytosis, the most serious adverse effects of deferiprone, are of interest.
Materials and Methods: In this retrospective population-based cohort study, paediatric thalassaemia patients receiving deferiprone were extracted from Hong Kong’s Clinical Data Analysis and Reporting System. The electronic patient charts were screened for serious and medically important adverse events (AE) related to chelation therapy using diagnosis codes, laboratory data and hospital admissions. Potential ADRs were assessed by reviewing concomitant medications, diagnoses and laboratory data and evaluated using a standardised causality assessment.
Results: Ninety-three patients contributing 153.7 person-years were included. A total of 509 AEs potentially related to deferiprone therapy were identified. After assessing causality, 30 ADRs in 20 patients were considered deferiprone-related and further analysed.
Incidence rates of neutropenia in mono- and combined therapy were 1.9 and 12.5 per 100 patient-years, respectively. The incidence rate ratio for neutropenia was 6.70 (95% CI 0.87-50.92; p=0.069). Severe neutropenia (agranulocytosis) was only observed in combined therapy. Other identified ADRs were severe arthropathy, elevated liver enzymes and mild thrombocytopenia. 54.2% of all ADRs occurred during the first three months of therapy.
Conclusion: In conclusion, the safety profile of deferiprone mono- and combination therapy observed in our paediatric cohort was in line with existing information, but children in simultaneous combined therapy experienced neutropenia more often than in monotherapy. Long-term safety studies and clinical trials are requested to better evaluate this risk.
