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.

Basic information

Original name

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

Authors

RANTA, Susanna, Jayashree MOTWANI, Jan BLATNÝ (203 Czech Republic, belonging to the institution), 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 and Christoph KOENIGS

Edition

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

Other information

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

30205 Hematology

Country of publisher

United States of America

Confidentiality degree

není předmětem státního či obchodního tajemství

References:

URL

Impact factor

Impact factor: 3.900 in 2022

RIV identification code

RIV/00216224:14110/23:00133414

Organization unit

Faculty of Medicine

DOI

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

UT WoS

001061475100001

Keywords in English

children; emicizumab; inhibitors; ITI; PUPs; survey

Abstract

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 and Christoph KOENIGS. Dilemmas on emicizumab in children with haemophilia A: A survey of strategies from PedNet centres. Haemophilia. HOBOKEN: Blackwell Science, 2023, vol. 29, No 5, p. 1291-1298. ISSN 1351-8216. Available from: 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 and Christoph KOENIGS. Dilemmas on emicizumab in children with haemophilia A: A survey of strategies from PedNet centres. \textit{Haemophilia}. HOBOKEN: Blackwell Science, 2023, vol.~29, No~5, p.~1291-1298. ISSN~1351-8216. Available from: https://dx.doi.org/10.1111/hae.14847.

Detailed Information on Publication Record