Compliance with Early Long-Term Prophylaxis Guidelines for Severe Hemophilia A

Saultier, Paul; Guillaume, Yves Claude; Demiguel, Virginie; Berger, Claire; Borel‐Derlon, Annie; Claeyssens, S.; Harroche, Annie; Oudot, Caroline; Rafowicz, Anne; Trossaërt, Marc; Wibaut, B.; Vinciguerra, C.; Boucékine, Mohamed; Baumstarck, Karine; Meunier, Sandrine; Calvez, Thierry; Chambost, Hérvè; PUPs, FranceCoag; Aouba, Achille; Legrand, Faézeh; Rothschild, Chantal; Torchet, M. F.; d’Oiron, Roseline; Lambert, Thierry; Laurian, Y.; Biernat, Jennifer; Goudemand, Jenny; Parquet, A.; Tintillier, Véronique; Assollant, Anne Durin; Négrier, Claude; Castet, Sabine; Guérin, V.; Huguenin, Yohann; Micheau, M; Ryman, Anne; Falaise, Céline; Thiercelin-Legrand, Marie Françoise; Fiks-Sigaud, M; Fouassier, Marc; Fressinaud, Édith; Voisin, Sophie; Faradji, A.; Lutz, Patrick; Briquel, M. E.; Frotscher, Birgit; Fimbel, Béatrice; Gruel, Yves; Guérois, C.; Régina, Sandra; Valentin, Jean Baptiste; Andreani, Christine Biron; Codine, P.; Donadio, D; Navarro, Robert; Rospide, Paola; Schved, Jean‐François; Collet, B; Derlon, A.; Gautier, Philippe; Bayart, Sophie; Guillet, Ben⊚ıt; Borg, Jeanne‐Yvonne; Dumesnil, C.; Normand, Charline; Schneider, Pascale; Tron, P; Vannier, Jean‐Pierre; Dutrillaux, F.; Volot, Fabienne; Gembara, Piotr; Marquès-Verdier, Alain; Adjaoud, Dalila; Barro, Claire; Pernod, Gilles; Polack, Ben⊚ıt; Pouzol, P; Ounnoughène, N.; Paugy, Patricia; Robert, Valérie; Stieltjes, N.; Bastenaire, Brigitte; Raucourt, Emmanuelle de; Peynet, J; Li-Thiao-Te, V.; Pautard, Brigitte; Béhar, C; Gorde, Stéphanie; Munzer, Martine; Lemoine, Elisabeth Benz; Macchi, Laurent; Lumley, L. De; Gaillard, Solange; Deville, Anne; Monpoux, Fabrice; Bertrand, Marie Anne; Pan‐Petesch, Brigitte; Hassoun, Abel; Coatmelec, B.; Beurrier, Philippe; Damay, M.; Moreau, Philippe; Pouille-Lievin, Odile; Schoepfer, Caroline; Tarral, E.; Bianchin, Monique; Nguyen, Joël; Pincemaille, O; Peter, Marie-Odile

doi:10.1016/j.jpeds.2021.02.071

The Journal of Pediatrics

2021

DOI: 10.1016/j.jpeds.2021.02.071

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Compliance with Early Long-Term Prophylaxis Guidelines for Severe Hemophilia A

Paul Saultier¹,

Yves Claude Guillaume²,

Virginie Demiguel³

et al.

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2023

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“…This is reflected in the survey by most centres using emicizumab as first line prophylaxis treatment with the opportunity to start significantly earlier than in traditional intravenous FVIII prophylaxis. Over the years, prophylaxis with FVIII concentrates has started earlier; in the PedNet centres, the median age for start of prophylaxis with coagulation factor concentrates in severe haemophilia A has decreased from 17.3 to 13.1 months over the last decades [9][10][11]. However, an even earlier start of FVIII prophylaxis is hampered by the need for intravenous injections, which is extremely challenging in very young children.…”

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confidence: 99%

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

Ranta,

Motwani,

Blatny

et al. 2023

Haemophilia

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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.

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confidence: 99%

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

Ranta,

Motwani,

Blatny

et al. 2023

Haemophilia

Self Cite

View full text Add to dashboard Cite

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Compliance with Early Long-Term Prophylaxis Guidelines for Severe Hemophilia A

Cited by 1 publication

References 24 publications

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

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

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