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

The result's identifiers

  • Result code in IS VaVaI

    <a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F65269705%3A_____%2F23%3A00078321" target="_blank" >RIV/65269705:_____/23:00078321 - isvavai.cz</a>

  • Alternative codes found

    RIV/00216224:14110/23:00133414 RIV/00216208:11130/23:10467129 RIV/00064203:_____/23:10467129

  • Result on the web

    <a href="https://onlinelibrary.wiley.com/doi/full/10.1111/hae.14847" target="_blank" >https://onlinelibrary.wiley.com/doi/full/10.1111/hae.14847</a>

  • DOI - Digital Object Identifier

    <a href="http://dx.doi.org/10.1111/hae.14847" target="_blank" >10.1111/hae.14847</a>

Alternative languages

  • Result language

    angličtina

  • Original language name

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

  • Original language description

    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.

  • Czech name

  • Czech description

Classification

  • Type

    J<sub>imp</sub> - Article in a specialist periodical, which is included in the Web of Science database

  • CEP classification

  • OECD FORD branch

    30205 - Hematology

Result continuities

  • Project

  • Continuities

    I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Others

  • Publication year

    2023

  • Confidentiality

    S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Data specific for result type

  • Name of the periodical

    Haemophilia

  • ISSN

    1351-8216

  • e-ISSN

    1365-2516

  • Volume of the periodical

    29

  • Issue of the periodical within the volume

    5

  • Country of publishing house

    US - UNITED STATES

  • Number of pages

    8

  • Pages from-to

    1291-1298

  • UT code for WoS article

    001061475100001

  • EID of the result in the Scopus database

    2-s2.0-85169166367