Dilemmas on emicizumab in children with haemophilia A: A survey
of strategies from PedNet centres

J 2023

Dilemmas on emicizumab in children with haemophilia A: A survey of strategies from PedNet centres

RANTA, Susanna, Jayashree MOTWANI, Jan BLATNÝ, Martina BUEHRLEN, Manuel CARCAO et. al.

Základní údaje

Originální název

Dilemmas on emicizumab in children with haemophilia A: A survey of strategies from PedNet centres

Autoři

RANTA, Susanna, Jayashree MOTWANI, Jan BLATNÝ (203 Česká republika, domácí), Martina BUEHRLEN, Manuel CARCAO, Herve CHAMBOST, Carmen ESCURIOLA, Kathelijn FISCHER, Mutlu KARTAL-KAESS, de Kovel MARLOES, Gili KENET, Christoph MALE, Beatrice NOLAN, Roseline OIRON, Martin OLIVIERI, Ester ZAPOTOCKA, Nadine G ANDERSSON a Christoph KOENIGS

Vydání

Haemophilia, HOBOKEN, Blackwell Science, 2023, 1351-8216

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30205 Hematology

Stát vydavatele

Spojené státy

Utajení

není předmětem státního či obchodního tajemství

Odkazy

URL

Impakt faktor

Impact factor: 3.900 v roce 2022

Kód RIV

RIV/00216224:14110/23:00133414

Organizační jednotka

Lékařská fakulta

DOI

http://dx.doi.org/10.1111/hae.14847

UT WoS

001061475100001

Klíčová slova anglicky

children; emicizumab; inhibitors; ITI; PUPs; survey

Štítky

14110321, rivok

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 2. 2. 2024 10:53, Mgr. Tereza Miškechová

Anotace

V originále

IntroductionHaemophilia 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.AimWe aimed to assess the current clinical practice on emicizumab prophylaxis within PedNet, a collaborative research platform for paediatricians treating children with haemophilia.MethodsAn 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.ResultsAll 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.ConclusionMost 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.

Citovat

RANTA, Susanna, Jayashree MOTWANI, Jan BLATNÝ, Martina BUEHRLEN, Manuel CARCAO, Herve CHAMBOST, Carmen ESCURIOLA, Kathelijn FISCHER, Mutlu KARTAL-KAESS, de Kovel MARLOES, Gili KENET, Christoph MALE, Beatrice NOLAN, Roseline OIRON, Martin OLIVIERI, Ester ZAPOTOCKA, Nadine G ANDERSSON a Christoph KOENIGS. Dilemmas on emicizumab in children with haemophilia A: A survey of strategies from PedNet centres. Haemophilia. HOBOKEN: Blackwell Science, 2023, roč. 29, č. 5, s. 1291-1298. ISSN 1351-8216. Dostupné z: https://dx.doi.org/10.1111/hae.14847.

@article{2369022,
   author = {Ranta, Susanna and Motwani, Jayashree and Blatný, Jan and Buehrlen, Martina and Carcao, Manuel and Chambost, Herve and Escuriola, Carmen and Fischer, Kathelijn and KartalandKaess, Mutlu and Marloes, de Kovel and Kenet, Gili and Male, Christoph and Nolan, Beatrice and Oiron, Roseline and Olivieri, Martin and Zapotocka, Ester and Andersson, Nadine G and Koenigs, Christoph},
   article_location = {HOBOKEN},
   article_number = {5},
   doi = {http://dx.doi.org/10.1111/hae.14847},
   keywords = {children; emicizumab; inhibitors; ITI; PUPs; survey},
   language = {eng},
   issn = {1351-8216},
   journal = {Haemophilia},
   title = {Dilemmas on emicizumab in children with haemophilia A: A survey of strategies from PedNet centres},
   url = {https://onlinelibrary.wiley.com/doi/10.1111/hae.14847},
   volume = {29},
   year = {2023}
}

TY  - JOUR
ID  - 2369022
AU  - Ranta, Susanna - Motwani, Jayashree - Blatný, Jan - Buehrlen, Martina - Carcao, Manuel - Chambost, Herve - Escuriola, Carmen - Fischer, Kathelijn - Kartal-Kaess, Mutlu - Marloes, de Kovel - Kenet, Gili - Male, Christoph - Nolan, Beatrice - Oiron, Roseline - Olivieri, Martin - Zapotocka, Ester - Andersson, Nadine G - Koenigs, Christoph
PY  - 2023
TI  - Dilemmas on emicizumab in children with haemophilia A: A survey of strategies from PedNet centres
JF  - Haemophilia
VL  - 29
IS  - 5
SP  - 1291-1298
EP  - 1291-1298
PB  - Blackwell Science
SN  - 13518216
KW  - children
KW  - emicizumab
KW  - inhibitors
KW  - ITI
KW  - PUPs
KW  - survey
UR  - https://onlinelibrary.wiley.com/doi/10.1111/hae.14847
N2  - IntroductionHaemophilia 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.AimWe aimed to assess the current clinical practice on emicizumab prophylaxis within PedNet, a collaborative research platform for paediatricians treating children with haemophilia.MethodsAn 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.ResultsAll 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.ConclusionMost 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.
ER  -

RANTA, Susanna, Jayashree MOTWANI, Jan BLATNÝ, Martina BUEHRLEN, Manuel CARCAO, Herve CHAMBOST, Carmen ESCURIOLA, Kathelijn FISCHER, Mutlu KARTAL-KAESS, de Kovel MARLOES, Gili KENET, Christoph MALE, Beatrice NOLAN, Roseline OIRON, Martin OLIVIERI, Ester ZAPOTOCKA, Nadine G ANDERSSON a Christoph KOENIGS. Dilemmas on emicizumab in children with haemophilia A: A survey of strategies from PedNet centres. \textit{Haemophilia}. HOBOKEN: Blackwell Science, 2023, roč.~29, č.~5, s.~1291-1298. ISSN~1351-8216. Dostupné z: https://dx.doi.org/10.1111/hae.14847.

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