Zobrazeno 1 - 10 of 99 pro vyhledávání: '"Marijke van den Berg"'
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Akademický článek
Inhibitor incidence in an unselected cohort of previously untreated patients with severe haemophilia B: a PedNet study
Autor: Christoph Male, Nadine G. Andersson, Anne Rafowicz, Ri Liesner, Karin Kurnik, Kathelijn Fischer, Helen Platokouki, Elena Santagostino, Hervé Chambost, Beatrice Nolan, Christoph Königs, Gili Kenet, Rolf Ljung, Marijke van den Berg, PedNet study group
Publikováno v: Haematologica, Vol 106, Iss 1 (2020)
The incidence of FIX inhibitors in severe hemophilia B (SHB) is not well defined. Frequencies of 3-5% have been reported but most studies to date were small, including patients with different severities, and without prospective follow-up for inhibito
Externí odkaz: https://doaj.org/article/ffae13c2f6ad42f4ad673b17a83bc52a
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4
Primary prophylaxis in children with severe haemophilia A and B-Implementation over the last 20 years as illustrated in real-world data in the PedNet cohorts
Autor: Rolf Ljung, H. Marijke Van den Berg, Marloes Stephanie De Kovel
Publikováno v: Haemophilia : the official journal of the World Federation of HemophiliaREFERENCES.
The prophylactic regimen in children with severe haemophilia is suggested in various publications and guidelines. Few data exist on its implementation in clinical practice.To investigate the implementation of primary prophylaxis based on real-life da
Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8db08e069c839f5071edfb472b1bd9a4
https://pubmed.ncbi.nlm.nih.gov/36571801
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Capturing Data in Rare Disease Registries to Support Regulatory Decision Making: A Survey Study Among Industry and Other Stakeholders
Autor: Patricia McGettigan, Sieta T. de Vries, Carla J. Jonker, Peter G. M. Mol, Arno W. Hoes, H. Marijke van den Berg
Publikováno v: Drug Safety. ADIS INT LTD
Drug Safety
Introduction In rare diseases, registry-based studies can be used to provide natural history data pre-approval and complement drug efficacy and/or safety knowledge post-approval. Objective The objective of this study was to investigate the opinion of
Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0c41e2e660712cce8e385c2113960c4f
https://doi.org/10.1007/s40264-021-01081-z
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7
Inhibitor development in previously untreated patients with severe haemophilia: A comparison of included patients and outcomes between a clinical study and a registry‐based study
Autor: H. Marijke van den Berg, Peter G. M. Mol, Arno W. Hoes, Carla J. Jonker, Katrien Oude Rengerink
Publikováno v: Haemophilia
Haemophilia, 26(5), 809-816. Wiley
Aim The aim of this study was to investigate whether a disease registry could serve as a suitable alternative to clinical studies to investigate safety of orphan drugs in children. Methods We used individual patient data from previously untreated pat
Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::79e64a1ea19bfbc09b81e736685858fc
https://doi.org/10.1111/hae.14100
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Inhibitor incidence in an unselected cohort of previously untreated patients with severe haemophilia B: a PedNet study
Autor: Christoph Male, Nadine G. Andersson, Anne Rafowicz, Ri Liesner, Karin Kurnik, Kathelijn Fischer, Helen Platokouki, Elena Santagostino, Hervé Chambost, Beatrice Nolan, Christoph Königs, Gili Kenet, Rolf Ljung, Marijke Van den Berg, null PedNet study group
Publikováno v: Haematologica
Haematologica, 2021, 106 (1), pp.123-129. ⟨10.3324/haematol.2019.239160⟩
Haematologica, Ferrata Storti Foundation, 2020, 106 (1), pp.123-129. ⟨10.3324/haematol.2019.239160⟩
The incidence of factor IX (FIX) inhibitors in severe hemophilia B (SHB) is not well defined. Frequencies of 3-5% have been reported but most studies to date have been small, including patients with different severities, and without prospective follo
Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::35bf5c91fefc3a728a5a1f42dbbb686d
https://hal.inrae.fr/hal-03329840
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10
ITI Treatment is not First-Choice Treatment in Children with Hemophilia A and Low-Responding Inhibitors: Evidence from a PedNet Study
Autor: Elena Santagostino, Christoph Königs, Jayashree Motwani, Roseline d'Oiron, Beatrice Nolan, Maria Elisa Mancuso, Torben Stamm Mikkelsen, H. Marijke van den Berg, Helen Platokouki
Publikováno v: Van Den Berg, H M, Mancuso, M E, Königs, C, D'Oiron, R, Platokouki, H, Mikkelsen, T S, Motwani, J, Nolan, B, Santagostino, E & European Pediatric Network for Haemophilia Management (PedNet) 2020, ' ITI Treatment is not First-Choice Treatment in Children with Hemophilia A and Low-Responding Inhibitors : Evidence from a PedNet Study ', Thrombosis and Haemostasis, vol. 120, no. 8, pp. 1166-1172 . https://doi.org/10.1055/s-0040-1713097
Background Limited data exist on the clinical impact of low-responding inhibitors and the requirement for immune tolerance induction (ITI) treatment to establish tolerance, reduce bleeding, and improve outcome. The aim of this article is to describe
Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::3ff7703ff67141bea6b524dbc9a3687d
https://pubmed.ncbi.nlm.nih.gov/32572865
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