MANCUSO, M. E., C. MALE, G. KENET, K. KAVAKLI, C. KONIGS, Jan BLATNÝ a K. FIJNVANDRAAT. Prophylaxis in children with haemophilia in an evolving treatment landscape. Haemophilia. Hoboken: Wiley-Blackwell, 2021, roč. 27, č. 6, s. 889-896. ISSN 1351-8216. Dostupné z: https://dx.doi.org/10.1111/hae.14412.
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Základní údaje
Originální název Prophylaxis in children with haemophilia in an evolving treatment landscape
Autoři MANCUSO, M. E., C. MALE, G. KENET, K. KAVAKLI, C. KONIGS, Jan BLATNÝ (203 Česká republika, domácí) a K. FIJNVANDRAAT (garant).
Vydání Haemophilia, Hoboken, Wiley-Blackwell, 2021, 1351-8216.
Další údaje
Originální jazyk angličtina
Typ výsledku Článek v odborném periodiku
Obor 30205 Hematology
Stát vydavatele Spojené státy
Utajení není předmětem státního či obchodního tajemství
WWW URL
Impakt faktor Impact factor: 4.263
Kód RIV RIV/00216224:14110/21:00124153
Organizační jednotka Lékařská fakulta
Doi http://dx.doi.org/10.1111/hae.14412
UT WoS 000697516900001
Klíčová slova anglicky arthropathy; children; EHL factor concentrate; haemophilia; non-replacement therapy; prophylaxis
Štítky 14110321, rivok
Příznaky Mezinárodní význam, Recenzováno
Změnil Změnila: Mgr. Tereza Miškechová, učo 341652. Změněno: 15. 2. 2022 09:51.
Anotace
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.
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