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

Autoři	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
Rok publikování	2023
Druh	Článek v odborném periodiku
Časopis / Zdroj	Haemophilia
Fakulta / Pracoviště MU	Lékařská fakulta
Citace
www	https://onlinelibrary.wiley.com/doi/10.1111/hae.14847
Doi	http://dx.doi.org/10.1111/hae.14847
Klíčová slova	children; emicizumab; inhibitors; ITI; PUPs; survey
Popis	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.