Development of a target concentration intervention to individualize paroxysmal nocturnal hemoglobinuria treatment with pegcetacoplan.
Publication year
2024Source
Annals of Hematology, 103, 7, (2024), pp. 2267-2272ISSN
Annotation
01 juli 2024
Publication type
Article / Letter to editor
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Organization
Pharmacy
Haematology
Paediatrics
Journal title
Annals of Hematology
Volume
vol. 103
Issue
iss. 7
Page start
p. 2267
Page end
p. 2272
Subject
Haematology - Radboud University Medical Center; Paediatrics - Radboud University Medical Center; Pharmacy - Radboud University Medical CenterAbstract
Pegcetacoplan (Aspaveli®/Empaveli™) is a factor C3 inhibitor that is approved for the treatment of paroxysmal nocturnal hemoglobinuria. An individualized dosing strategy might be useful to improve patient-friendliness and cost-effectiveness of this very expensive drug. Therefore, the aim of this study was to develop an individualized treatment regimen for pegcetacoplan based on the pharmacokinetic-pharmacodynamic data of the manufacturer. We conducted a clinical trial simulation with the approved dosing regimen of 1080 mg twice-weekly and a target concentration intervention-based dosing regimen in patients with and without prior eculizumab use. For eculizumab-naïve patients, the target concentration intervention-based dosing regimen resulted in a comparable fraction of patients with LDH normalization (LDH < 226 U/L) and hemoglobulin normalization (> 12 g/dL) compared to the approved regimen (LDH 50.2% and 50.0% respectively and hemoglobulin 45.6% and 44.4%). A modest dose reduction of ~ 5% was possible with target concentration intervention-based dosing. An intensified dosing interval was necessary in 2.3% of the patients however an interval prolongation was possible in 28.2% of the patients. Similar results were obtained for patients prior treated with eculizumab. In this study we show the potential of an individualized dosing regimen of pegcetacoplan with can improve patient friendliness in approximately 30% of the patients and improve therapy in approximately 2% of the patients at slightly reduced costs.
This item appears in the following Collection(s)
- Academic publications [246515]
- Electronic publications [134102]
- Faculty of Medical Sciences [93308]
- Open Access publications [107627]
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