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How to achieve full prophylaxis in young boys with severe haemophilia A: different regimens and their effect on early bleeding and venous access

Nijdam, A.; Kurnik, K.; Liesner, R.; Ljung, Rolf LU ; Nolan, B.; Petrini, P. and Fischer, K. (2015) In Haemophilia 21(4). p.444-450
Abstract
To facilitate early prophylaxis, step-up regimens starting prophylaxis with infusions 1xweek(-1) were introduced. Choice of initial regimen may affect outcome. This study aims to classify initial prophylactic regimens and compare them on short-term outcome. From the European Paediatric Network for Haemophilia Management' (PedNet) registry, patients with severe haemophilia A without inhibitors, born 2000-2012, receiving prophylaxis were included. Treatment centres were classified according to the initial frequency of prophylactic infusions and the age at reaching infusions >= 3 x week(-1). Bleeding, and central venous access device (CVAD) use were compared at age 4 years. In 21 centres with 363 patients, three regimens were identified:... (More)
To facilitate early prophylaxis, step-up regimens starting prophylaxis with infusions 1xweek(-1) were introduced. Choice of initial regimen may affect outcome. This study aims to classify initial prophylactic regimens and compare them on short-term outcome. From the European Paediatric Network for Haemophilia Management' (PedNet) registry, patients with severe haemophilia A without inhibitors, born 2000-2012, receiving prophylaxis were included. Treatment centres were classified according to the initial frequency of prophylactic infusions and the age at reaching infusions >= 3 x week(-1). Bleeding, and central venous access device (CVAD) use were compared at age 4 years. In 21 centres with 363 patients, three regimens were identified: (i) start prophylaxis with >= 3 x week(-1) infusions before age three (full: 19% of centres, 18% of patients); (ii) start 1-2 x week(-1), increasing frequency as soon as possible (asap), reaching >= 3 x week(-1) before age three (43% of centres, 36% of patients); (iii) start 1-2 x week(-1), increasing frequency according to bleeding (phenotype), reaching >= 3 x week(-1) after age three (38% of centres, 46% of patients). Prophylaxis was started at median 1.2 years on the full and asap regimen vs 1.8 years on the phenotype regimen. Complete prevention of joint bleeds was most effective on the full regimen (32% full vs. 27% asap and 8% phenotype), though at the cost of using most CVADs (88% full vs. 34% asap and 22% phenotype). The three prophylaxis regimens identified had different effects on early bleeding and CVAD use. This classification provides the first step towards establishing the optimum prophylactic regimen. (Less)
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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
bleeding, central venous catheters, children, haemophilia A, prophylaxis, step-up regimen
in
Haemophilia
volume
21
issue
4
pages
444 - 450
publisher
Federation of European Neuroscience Societies and Blackwell Publishing Ltd
external identifiers
  • wos:000356875600022
  • scopus:84931957417
ISSN
1351-8216
DOI
10.1111/hae.12613
language
English
LU publication?
yes
id
6aede1a3-3af9-4102-af01-a771f7f4e405 (old id 7791064)
date added to LUP
2015-09-01 16:10:16
date last changed
2017-11-12 03:03:51
@article{6aede1a3-3af9-4102-af01-a771f7f4e405,
  abstract     = {To facilitate early prophylaxis, step-up regimens starting prophylaxis with infusions 1xweek(-1) were introduced. Choice of initial regimen may affect outcome. This study aims to classify initial prophylactic regimens and compare them on short-term outcome. From the European Paediatric Network for Haemophilia Management' (PedNet) registry, patients with severe haemophilia A without inhibitors, born 2000-2012, receiving prophylaxis were included. Treatment centres were classified according to the initial frequency of prophylactic infusions and the age at reaching infusions >= 3 x week(-1). Bleeding, and central venous access device (CVAD) use were compared at age 4 years. In 21 centres with 363 patients, three regimens were identified: (i) start prophylaxis with >= 3 x week(-1) infusions before age three (full: 19% of centres, 18% of patients); (ii) start 1-2 x week(-1), increasing frequency as soon as possible (asap), reaching >= 3 x week(-1) before age three (43% of centres, 36% of patients); (iii) start 1-2 x week(-1), increasing frequency according to bleeding (phenotype), reaching >= 3 x week(-1) after age three (38% of centres, 46% of patients). Prophylaxis was started at median 1.2 years on the full and asap regimen vs 1.8 years on the phenotype regimen. Complete prevention of joint bleeds was most effective on the full regimen (32% full vs. 27% asap and 8% phenotype), though at the cost of using most CVADs (88% full vs. 34% asap and 22% phenotype). The three prophylaxis regimens identified had different effects on early bleeding and CVAD use. This classification provides the first step towards establishing the optimum prophylactic regimen.},
  author       = {Nijdam, A. and Kurnik, K. and Liesner, R. and Ljung, Rolf and Nolan, B. and Petrini, P. and Fischer, K.},
  issn         = {1351-8216},
  keyword      = {bleeding,central venous catheters,children,haemophilia A,prophylaxis,step-up regimen},
  language     = {eng},
  number       = {4},
  pages        = {444--450},
  publisher    = {Federation of European Neuroscience Societies and Blackwell Publishing Ltd},
  series       = {Haemophilia},
  title        = {How to achieve full prophylaxis in young boys with severe haemophilia A: different regimens and their effect on early bleeding and venous access},
  url          = {http://dx.doi.org/10.1111/hae.12613},
  volume       = {21},
  year         = {2015},
}