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Dilemmas on emicizumab in children with haemophilia A: A survey of strategies from PedNet centres
S. Ranta, J. Motwani, J. Blatny, M. Bührlen, M. Carcao, H. Chambost, C. Escuriola, K. Fischer, M. Kartal-Kaess, M. de Kovel, G. Kenet, C. Male, B. Nolan, R. d'Oiron, M. Olivieri, E. Zapotocka, NG. Andersson, C. Königs
Jazyk angličtina Země Anglie, Velká Británie
Typ dokumentu časopisecké články
Grantová podpora
FNBr 65269705
PedNet Hemophilia Research Foundation and MH CZ-DRO
PubMed
37647211
DOI
10.1111/hae.14847
Knihovny.cz E-zdroje
- MeSH
- dítě MeSH
- elektronika MeSH
- hemofilie A * farmakoterapie MeSH
- humanizované monoklonální protilátky terapeutické užití MeSH
- kojenec MeSH
- lidé MeSH
- protilátky bispecifické * terapeutické užití MeSH
- Check Tag
- dítě MeSH
- kojenec MeSH
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
INTRODUCTION: Haemophilia A care has changed with the introduction of emicizumab. Experience on the youngest children is still scarce and clinical practice varies between haemophilia treatment centres. AIM: We aimed to assess the current clinical practice on emicizumab prophylaxis within PedNet, a collaborative research platform for paediatricians treating children with haemophilia. METHODS: An electronic survey was sent to all PedNet members (n = 32) between October 2022 and February 2023. The survey included questions on the availability of emicizumab, on the practice of initiating prophylaxis in previously untreated or minimally treated patients (PUPs or MTPs) and emicizumab use in patients with or without inhibitors. RESULTS: All but four centres (28/32; 88%) responded. Emicizumab was available in clinical practice in 25/28 centres (89%), and in 3/28 for selected patients only (e.g. with inhibitors). Emicizumab was the preferred choice for prophylaxis in PUPs or MTPs in 20/25 centres; most (85%) started emicizumab prophylaxis before 1 year of age (30% before 6 months of age) and without concomitant FVIII (16/20; 80%). After the loading dose, 13/28 centres administered the recommended dosing, while the others adjusted the interval of injections to give whole vials. In inhibitor patients, the use of emicizumab during ITI was common, with low-dose ITI being the preferred protocol. CONCLUSION: Most centres choose to initiate prophylaxis with emicizumab before 12 months of age and without concomitant FVIII. In inhibitor patients, ITI is mostly given in addition to emicizumab, but there was no common practice on how to proceed after successful ITI.
Birmingham Children's Hospital Birmingham UK
Children's Coagulation Centre Children's Health Ireland at Crumlin Dublin Ireland
Department of Paediatrics and Adolescent Medicine Goethe University Frankfurt Frankfurt Germany
Department of Paediatrics Medical University of Vienna Vienna Austria
Department of Pediatrics Klinikum Bremen Mitte Bremen Germany
Haemophilie Zentrum Rhein Main HZRM Mörfelden Walldorf Germany
PedNet Haemophilia Research Foundation Baarn The Netherlands
Citace poskytuje Crossref.org
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- $a INTRODUCTION: Haemophilia A care has changed with the introduction of emicizumab. Experience on the youngest children is still scarce and clinical practice varies between haemophilia treatment centres. AIM: We aimed to assess the current clinical practice on emicizumab prophylaxis within PedNet, a collaborative research platform for paediatricians treating children with haemophilia. METHODS: An electronic survey was sent to all PedNet members (n = 32) between October 2022 and February 2023. The survey included questions on the availability of emicizumab, on the practice of initiating prophylaxis in previously untreated or minimally treated patients (PUPs or MTPs) and emicizumab use in patients with or without inhibitors. RESULTS: All but four centres (28/32; 88%) responded. Emicizumab was available in clinical practice in 25/28 centres (89%), and in 3/28 for selected patients only (e.g. with inhibitors). Emicizumab was the preferred choice for prophylaxis in PUPs or MTPs in 20/25 centres; most (85%) started emicizumab prophylaxis before 1 year of age (30% before 6 months of age) and without concomitant FVIII (16/20; 80%). After the loading dose, 13/28 centres administered the recommended dosing, while the others adjusted the interval of injections to give whole vials. In inhibitor patients, the use of emicizumab during ITI was common, with low-dose ITI being the preferred protocol. CONCLUSION: Most centres choose to initiate prophylaxis with emicizumab before 12 months of age and without concomitant FVIII. In inhibitor patients, ITI is mostly given in addition to emicizumab, but there was no common practice on how to proceed after successful ITI.
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