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Intracranial haemorrhage in children and adolescents with severe haemophilia A or B - the impact of prophylactic treatment

Gretenkort Andersson, Nadine LU ; Auerswald, Günter ; Barnes, Chris ; Carcao, Manuel ; Dunn, Amy L. ; Fijnvandraat, Karin ; Hoffmann, Marianne ; Kavakli, Kaan ; Kenet, Gili and Kobelt, Rainer , et al. (2017) In British Journal of Haematology 179(2). p.298-307
Abstract

The discussion of prophylactic therapy in haemophilia is largely focused on joint outcomes. The impact of prophylactic therapy on intracranial haemorrhage (ICH) is less known. This study aimed to analyse ICH in children with haemophilia, with a focus on different prophylaxis regimens and sequelae of ICH. We conducted a multicentre retrospective and prospective study that included 33 haemophilia centres from 20 countries. Inclusion criteria were children and adolescents born between 1993 and 2014, with severe haemophilia A or B without inhibitors. Participants were categorized by prophylaxis regimen: full, partial or none, based on dose and dose frequency of regular infusions. The cohort study included 1515 children: 29 cases of ICH over... (More)

The discussion of prophylactic therapy in haemophilia is largely focused on joint outcomes. The impact of prophylactic therapy on intracranial haemorrhage (ICH) is less known. This study aimed to analyse ICH in children with haemophilia, with a focus on different prophylaxis regimens and sequelae of ICH. We conducted a multicentre retrospective and prospective study that included 33 haemophilia centres from 20 countries. Inclusion criteria were children and adolescents born between 1993 and 2014, with severe haemophilia A or B without inhibitors. Participants were categorized by prophylaxis regimen: full, partial or none, based on dose and dose frequency of regular infusions. The cohort study included 1515 children: 29 cases of ICH over 8038 patient years were reported. The incidence of ICH in the prophylaxis group, 0·00033 cases of ICH/patient year, was significantly lower compared to the no prophylaxis group, 0·017 cases of ICH/patient year (RR 50·06; P < 0·001) and the partial prophylaxis group, 0·0050 cases of ICH/patient year (RR 14·92; P = 0·007). In the on-demand-group, 8% (2/24) children with ICH died and 33% had long-term sequelae, including intellectual and behavioural problems, paresis and epilepsy. Children on regular, frequent prophylaxis have a low risk of ICH compared to those using non-frequent or no prophylaxis.

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organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
Children, Factor IX, Factor VIII, Haemophilia A, Haemophilia B
in
British Journal of Haematology
volume
179
issue
2
pages
298 - 307
publisher
Wiley-Blackwell
external identifiers
  • scopus:85023170005
  • pmid:28699675
  • wos:000412835500015
ISSN
0007-1048
DOI
10.1111/bjh.14844
language
English
LU publication?
yes
id
c455e26d-9218-46f0-a051-a1ee5e24fed5
date added to LUP
2017-08-23 15:25:59
date last changed
2024-04-14 16:17:52
@article{c455e26d-9218-46f0-a051-a1ee5e24fed5,
  abstract     = {{<p>The discussion of prophylactic therapy in haemophilia is largely focused on joint outcomes. The impact of prophylactic therapy on intracranial haemorrhage (ICH) is less known. This study aimed to analyse ICH in children with haemophilia, with a focus on different prophylaxis regimens and sequelae of ICH. We conducted a multicentre retrospective and prospective study that included 33 haemophilia centres from 20 countries. Inclusion criteria were children and adolescents born between 1993 and 2014, with severe haemophilia A or B without inhibitors. Participants were categorized by prophylaxis regimen: full, partial or none, based on dose and dose frequency of regular infusions. The cohort study included 1515 children: 29 cases of ICH over 8038 patient years were reported. The incidence of ICH in the prophylaxis group, 0·00033 cases of ICH/patient year, was significantly lower compared to the no prophylaxis group, 0·017 cases of ICH/patient year (RR 50·06; P &lt; 0·001) and the partial prophylaxis group, 0·0050 cases of ICH/patient year (RR 14·92; P = 0·007). In the on-demand-group, 8% (2/24) children with ICH died and 33% had long-term sequelae, including intellectual and behavioural problems, paresis and epilepsy. Children on regular, frequent prophylaxis have a low risk of ICH compared to those using non-frequent or no prophylaxis.</p>}},
  author       = {{Gretenkort Andersson, Nadine and Auerswald, Günter and Barnes, Chris and Carcao, Manuel and Dunn, Amy L. and Fijnvandraat, Karin and Hoffmann, Marianne and Kavakli, Kaan and Kenet, Gili and Kobelt, Rainer and Kurnik, Karin and Liesner, Ri and Mäkipernaa, Anne and Manco-Johnson, Marilyn J and Mancuso, Maria Elisa and Molinari, Angelo Claudio and Nolan, Beatrice and Perez Garrido, Rosario and Petrini, Pia and Platokouki, Helen E. and Shapiro, Amy D. and Wu, Runhui and Ljung, Rolf}},
  issn         = {{0007-1048}},
  keywords     = {{Children; Factor IX; Factor VIII; Haemophilia A; Haemophilia B}},
  language     = {{eng}},
  number       = {{2}},
  pages        = {{298--307}},
  publisher    = {{Wiley-Blackwell}},
  series       = {{British Journal of Haematology}},
  title        = {{Intracranial haemorrhage in children and adolescents with severe haemophilia A or B - the impact of prophylactic treatment}},
  url          = {{http://dx.doi.org/10.1111/bjh.14844}},
  doi          = {{10.1111/bjh.14844}},
  volume       = {{179}},
  year         = {{2017}},
}