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. 2023 Sep;29(5):1291-1298.
doi: 10.1111/hae.14847. Epub 2023 Aug 30.

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

Susanna Ranta  1 , Jayashree Motwani  2 , Jan Blatny  3 , Martina Bührlen  4 , Manuel Carcao  5 , Hervé Chambost  6 , Carmen Escuriola  7 , Kathelijn Fischer  8 , Mutlu Kartal-Kaess  9 , Marloes de Kovel  10 , Gili Kenet  11 , Christoph Male  12 , Beatrice Nolan  13 , Roseline d'Oiron  14 , Martin Olivieri  15 , Ester Zapotocka  16 , Nadine G Andersson  17 , Christoph Königs  18
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Free article

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

Susanna Ranta et al. Haemophilia. 2023 Sep.
Free article

Abstract

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.

Keywords: ITI; PUPs; children; emicizumab; inhibitors; survey.

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