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Prophylaxis in children with haemophilia in an evolving treatment landscape

The result's identifiers

  • Result code in IS VaVaI

    <a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F65269705%3A_____%2F21%3A00074775" target="_blank" >RIV/65269705:_____/21:00074775 - isvavai.cz</a>

  • Alternative codes found

    RIV/00216224:14110/21:00124153

  • Result on the web

    <a href="https://onlinelibrary.wiley.com/doi/10.1111/hae.14412" target="_blank" >https://onlinelibrary.wiley.com/doi/10.1111/hae.14412</a>

  • DOI - Digital Object Identifier

    <a href="http://dx.doi.org/10.1111/hae.14412" target="_blank" >10.1111/hae.14412</a>

Alternative languages

  • Result language

    angličtina

  • Original language name

    Prophylaxis in children with haemophilia in an evolving treatment landscape

  • Original language description

    Introduction For children with haemophilia, early initiation of prophylaxis is crucial to prevent life-threatening bleeds and maintain joint health throughout life. Options for prophylaxis have recently increased from replacement therapy with standard or extended half-life coagulation factor products to include other haemostasis products, such as the non-replacement therapy emicizumab. Aim To review key factors that determine the choice of prophylaxis in young children. Methods Key clinical questions on the implementation of prophylaxis for haemophilia in children were identified and PubMed was searched for evidence supporting guidance on the implementation of prophylaxis. Results The results of the literature search and the practical experience of the authors were used to build consensus on when to start prophylaxis, the pros and cons of the products available to guide the choice of product, and practical aspects of starting prophylaxis to guide the choice of regimen. Conclusions In this era of increasing therapeutic choices, available information about the range of treatment options must be considered when initiating prophylaxis in young children. Parents or care givers must be sufficiently informed to allow informed shared decision making. Although plentiful data and clinical experience have been gathered on prophylaxis with clotting factor replacement therapy, its use in young children brings practical challenges, such as the need for intravenous administration. In contrast, our relatively brief experience and limited data with subcutaneously administered non-replacement therapy (i.e., emicizumab) in this patient group imply that starting emicizumab prophylaxis in young children requires careful consideration, despite the more convenient route of administration.

  • 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

    N - Vyzkumna aktivita podporovana z neverejnych zdroju

Others

  • Publication year

    2021

  • 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

  • Volume of the periodical

    27

  • Issue of the periodical within the volume

    6

  • Country of publishing house

    US - UNITED STATES

  • Number of pages

    8

  • Pages from-to

    889-896

  • UT code for WoS article

    000697516900001

  • EID of the result in the Scopus database

    2-s2.0-85115248492