Prophylaxis in children with haemophilia in an evolving
treatment landscape

J 2021

Prophylaxis in children with haemophilia in an evolving treatment landscape

MANCUSO, M. E., C. MALE, G. KENET, K. KAVAKLI, C. KONIGS et. al.

Basic information

Original name

Prophylaxis in children with haemophilia in an evolving treatment landscape

Authors

MANCUSO, M. E., C. MALE, G. KENET, K. KAVAKLI, C. KONIGS, Jan BLATNÝ (203 Czech Republic, belonging to the institution) and K. FIJNVANDRAAT (guarantor)

Edition

Haemophilia, Hoboken, Wiley-Blackwell, 2021, 1351-8216

Other information

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

30205 Hematology

Country of publisher

United States of America

Confidentiality degree

není předmětem státního či obchodního tajemství

References:

URL

Impact factor

Impact factor: 4.263

RIV identification code

RIV/00216224:14110/21:00124153

Organization unit

Faculty of Medicine

DOI

http://dx.doi.org/10.1111/hae.14412

UT WoS

000697516900001

Keywords in English

arthropathy; children; EHL factor concentrate; haemophilia; non-replacement therapy; prophylaxis

Abstract

V originále

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.

Citovat

MANCUSO, M. E., C. MALE, G. KENET, K. KAVAKLI, C. KONIGS, Jan BLATNÝ and K. FIJNVANDRAAT. Prophylaxis in children with haemophilia in an evolving treatment landscape. Haemophilia. Hoboken: Wiley-Blackwell, 2021, vol. 27, No 6, p. 889-896. ISSN 1351-8216. Available from: https://dx.doi.org/10.1111/hae.14412.

@article{1834304,
   author = {Mancuso, M. E. and Male, C. and Kenet, G. and Kavakli, K. and Konigs, C. and Blatný, Jan and Fijnvandraat, K.},
   article_location = {Hoboken},
   article_number = {6},
   doi = {http://dx.doi.org/10.1111/hae.14412},
   keywords = {arthropathy; children; EHL factor concentrate; haemophilia; non-replacement therapy; prophylaxis},
   language = {eng},
   issn = {1351-8216},
   journal = {Haemophilia},
   title = {Prophylaxis in children with haemophilia in an evolving treatment landscape},
   url = {https://onlinelibrary.wiley.com/doi/10.1111/hae.14412},
   volume = {27},
   year = {2021}
}

TY  - JOUR
ID  - 1834304
AU  - Mancuso, M. E. - Male, C. - Kenet, G. - Kavakli, K. - Konigs, C. - Blatný, Jan - Fijnvandraat, K.
PY  - 2021
TI  - Prophylaxis in children with haemophilia in an evolving treatment landscape
JF  - Haemophilia
VL  - 27
IS  - 6
SP  - 889-896
EP  - 889-896
PB  - Wiley-Blackwell
SN  - 13518216
KW  - arthropathy
KW  - children
KW  - EHL factor concentrate
KW  - haemophilia
KW  - non-replacement therapy
KW  - prophylaxis
UR  - https://onlinelibrary.wiley.com/doi/10.1111/hae.14412
N2  - 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.
ER  -

MANCUSO, M. E., C. MALE, G. KENET, K. KAVAKLI, C. KONIGS, Jan BLATNÝ and K. FIJNVANDRAAT. Prophylaxis in children with haemophilia in an evolving treatment landscape. \textit{Haemophilia}. Hoboken: Wiley-Blackwell, 2021, vol.~27, No~6, p.~889-896. ISSN~1351-8216. Available from: https://dx.doi.org/10.1111/hae.14412.

Detailed Information on Publication Record