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Methods for individualising factor VIII dosing in prophylaxis

Ar, Muhlis Cem; Vaide, Ines; Berntorp, Erik LU and Bjorkman, Sven (2014) In European Journal of Haematology 93. p.16-20
Abstract
Haemophilia A is a sex-linked disorder characterised chiefly by recurrent, spontaneous joint and muscle bleedings resulting from deficiency of factor VIII (FVIII). Recurrent joint bleeds result in haemophilic arthropathy. Unless treated with factor replacement therapy, many patients with severe haemophilia become disabled. The first clinical evidence favouring prophylaxis originated from the studies in Sweden and the Netherlands in the 1960s. Later on, it was shown that prophylaxis could prevent arthropathy, if started early in life, or slow its progression in adults with established arthropathy. The optimal dosing of FVIII in long-term prophylaxis has still not been determined, and there is growing evidence that the dose and frequency of... (More)
Haemophilia A is a sex-linked disorder characterised chiefly by recurrent, spontaneous joint and muscle bleedings resulting from deficiency of factor VIII (FVIII). Recurrent joint bleeds result in haemophilic arthropathy. Unless treated with factor replacement therapy, many patients with severe haemophilia become disabled. The first clinical evidence favouring prophylaxis originated from the studies in Sweden and the Netherlands in the 1960s. Later on, it was shown that prophylaxis could prevent arthropathy, if started early in life, or slow its progression in adults with established arthropathy. The optimal dosing of FVIII in long-term prophylaxis has still not been determined, and there is growing evidence that the dose and frequency of FVIII should be individualised. We conducted a systematic search of PubMed to identify all relevant articles on FVIII prophylaxis in severe haemophilia A. We focused on articles with detailed information about individualisation of prophylaxis. Long-term prophylaxis in haemophilia was introduced in Sweden in the late 1950s. However, standard prophylactic regimens may not be appropriate for all patients with severe haemophilia. Factors such as age, joint status, co-morbidities and differences in pharmacokinetics lead to interindividual variation in factor requirement. Dose tailoring of FVIII by clinical outcome was first described in 1994. Since then, several dose-finding studies questioned the necessity to maintain preinfusion levels of FVIII above 1%. Individualising prophylaxis by dose tailoring is now recommended. Each country should adopt policies for individualising prophylaxis in patients with severe haemophilia. This would lead to a better distribution of the available source of factor concentrates. (Less)
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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
prophylaxis, haemophilia, pharmacokinetics, treatment tailoring
in
European Journal of Haematology
volume
93
pages
16 - 20
publisher
Wiley-Blackwell
external identifiers
  • wos:000338019900003
  • scopus:84902957622
ISSN
1600-0609
DOI
10.1111/ejh.12370
language
English
LU publication?
yes
id
db34a1e8-ae83-428b-97bf-8c22cb76a561 (old id 4608851)
date added to LUP
2014-09-01 07:37:58
date last changed
2017-11-05 03:25:36
@article{db34a1e8-ae83-428b-97bf-8c22cb76a561,
  abstract     = {Haemophilia A is a sex-linked disorder characterised chiefly by recurrent, spontaneous joint and muscle bleedings resulting from deficiency of factor VIII (FVIII). Recurrent joint bleeds result in haemophilic arthropathy. Unless treated with factor replacement therapy, many patients with severe haemophilia become disabled. The first clinical evidence favouring prophylaxis originated from the studies in Sweden and the Netherlands in the 1960s. Later on, it was shown that prophylaxis could prevent arthropathy, if started early in life, or slow its progression in adults with established arthropathy. The optimal dosing of FVIII in long-term prophylaxis has still not been determined, and there is growing evidence that the dose and frequency of FVIII should be individualised. We conducted a systematic search of PubMed to identify all relevant articles on FVIII prophylaxis in severe haemophilia A. We focused on articles with detailed information about individualisation of prophylaxis. Long-term prophylaxis in haemophilia was introduced in Sweden in the late 1950s. However, standard prophylactic regimens may not be appropriate for all patients with severe haemophilia. Factors such as age, joint status, co-morbidities and differences in pharmacokinetics lead to interindividual variation in factor requirement. Dose tailoring of FVIII by clinical outcome was first described in 1994. Since then, several dose-finding studies questioned the necessity to maintain preinfusion levels of FVIII above 1%. Individualising prophylaxis by dose tailoring is now recommended. Each country should adopt policies for individualising prophylaxis in patients with severe haemophilia. This would lead to a better distribution of the available source of factor concentrates.},
  author       = {Ar, Muhlis Cem and Vaide, Ines and Berntorp, Erik and Bjorkman, Sven},
  issn         = {1600-0609},
  keyword      = {prophylaxis,haemophilia,pharmacokinetics,treatment tailoring},
  language     = {eng},
  pages        = {16--20},
  publisher    = {Wiley-Blackwell},
  series       = {European Journal of Haematology},
  title        = {Methods for individualising factor VIII dosing in prophylaxis},
  url          = {http://dx.doi.org/10.1111/ejh.12370},
  volume       = {93},
  year         = {2014},
}