Population pharmacokinetics and dosing recommendations for the use of deferiprone in children younger than 6 years

Bellanti, Francesco; Vecchio, Giovanni Carlo Del; Putti, Maria Caterina; Maggio, Aurelio; Filosa, Aldo; Cosmi, Carlo; Mangiarini, Laura; Spino, Michael; Connelly, John T.; Ceci, Adriana; Pasqua, Oscar Della

doi:10.1111/bcp.13134

Cited by 8 publications

(3 citation statements)

References 27 publications

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“…12,13 This is partly explained by inaccurate estimation of covariateparameter correlations and in some cases because of missing or misspecified covariates on key parameters such as clearance and volume of distribution. 14,15 Consequently, it is rather surprising that despite the availability of different methods to integrate prior knowledge for the analysis of such sparse and unbalanced data, 16,17 and for the optimisation of the study design, [18][19][20] their uptake in clinical research, and, particularly, in case of rare diseases, remains very low.…”

Section: Accepted Manuscriptmentioning

confidence: 99%

Use of prior knowledge and extrapolation in paediatric drug development: A case study with deferasirox

Borella

Oosterholt

Magni

et al. 2019

European Journal of Pharmaceutical Sciences

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Section: Accepted Manuscriptmentioning

confidence: 99%

Use of prior knowledge and extrapolation in paediatric drug development: A case study with deferasirox

Borella

Oosterholt

Magni

et al. 2019

European Journal of Pharmaceutical Sciences

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“…Alternatively, if MRI is not available or applicable, serum ferritin levels of ≥1000 lg/l and/or transferrin saturation levels ≥75% have been used as a starting point for chelation therapy. While chelating drugs are not registered yet for children younger than 2 years old (deferiprone <6 years), results of recent studies in younger children with transfusion-related iron overload are promising (Roggero et al, 2009;Berdoukas et al, 2013;Marsella & Borgna-Pignatti, 2014;Origa et al, 2016;Bellanti et al, 2017;Taher & Saliba, 2017;Elalfy et al, 2018).…”

Section: Iron Overloadmentioning

confidence: 99%

How I manage children with Diamond‐Blackfan anaemia

Bartels

Bierings

2018

Br J Haematol

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Summary Diamond‐Blackfan anaemia ( DBA ) is a rare inherited marrow failure disorder, characterized by hypoplastic anaemia, congenital anomalies and a predisposition to cancer as a result of ribosomal dysfunction. Historically, treatment is based on glucocorticoids and/or blood transfusions, which is accompanied by significant toxicity and long‐term sequelae. Currently, stem cell transplantation is the only curative option for the haematological DBA phenotype. Whereas this procedure has been quite successful in the last decade in selected patients, novel therapies and biological insights are still warranted to improve clinical care for all DBA patients. In addition to paediatric haematologists, other physicians (e.g. endocrinologist, gynaecologist) should ideally be involved in the care of this chronic condition from an early age, to improve lifelong management of haematological and non‐haematological symptoms, and screen for DBA ‐associated malignancies. Here we provide an overview of current knowledge and recommendations for the day‐to‐day care of DBA patients.

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“…It has been widely used for four decades despite its limitations (suboptimal potential for compliance due to mode of administration, as well as side effects, especially, in the context of lower iron load) [ 7 , 15 ]. DFP has the advantage of being given orally, but is indicated as second line treatment [ 16 ]. The newest oral chelator, DFX, is licensed as first line treatment for children over 6 years of age, and as first line treatment in USA—but second line treatment in Europe—for children of younger age (2–6 years old) [ 6 ].…”

Section: Introductionmentioning

confidence: 99%

Use of Deferasirox Film-Coated Tablets in Pediatric Patients with Transfusion Dependent Thalassemia: A Single Center Experience

Adramerina

Printza

Hatzipantelis

et al. 2022

Biology

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Thalassemic syndromes are characterized by clinical heterogenicity. For severe disease forms, lifelong blood transfusions remain the mainstay of therapy, while iron overload monitoring and adequate chelation treatment are required in order to ensure effective disease management. Compared to previous chelators, the new deferasirox film-coated tablet (DFX FCT) is considered to offer a more convenient and well-tolerated treatment scheme, aiming at better treatment-related and patient-related outcomes. The present study’s objective was to prospectively evaluate the safety and efficacy of DFX FCT in children and adolescents with transfusion-dependent thalassemia. Data collected included patient demographics, hematology and biochemistry laboratory work up, magnetic resonance imaging of heart and liver for iron load, as well as ophthalmological and audiological examination prior to and a year following drug administration. Study results confirmed DFX FCT safety in older children in a manner similar to adults, but demonstrated increased frequency of adverse events in younger patients, mainly, involving liver function. With regards to efficacy, study results confirmed the preventive role of DFX FCT in iron loading of liver and heart, however, higher doses than generally recommended were required in order to ensure adequate chelation.

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Population pharmacokinetics and dosing recommendations for the use of deferiprone in children younger than 6 years

Abstract: Based on the current findings, a dosing regimen of 25 mg kg t.i.d. is recommended in children aged <6 years, with the possibility of titration up to 33.3 mg kg t.i.d.

Cited by 8 publications

References 27 publications

Use of prior knowledge and extrapolation in paediatric drug development: A case study with deferasirox

Use of prior knowledge and extrapolation in paediatric drug development: A case study with deferasirox

How I manage children with Diamond‐Blackfan anaemia

Use of Deferasirox Film-Coated Tablets in Pediatric Patients with Transfusion Dependent Thalassemia: A Single Center Experience

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