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Strategies for inhibitor treatment and costs in the short and long term: a critical evaluation of recent clinical studies

Baghaipour, Mohammad Reza and Steen Carlsson, Katarina LU (2015) In European Journal of Haematology 94. p.30-37
Abstract
One important complication of patients with severe haemophilia A is the formation of inhibitory antibodies to factor VIII (FVIII). Immune tolerance induction (ITI) is the treatment of choice for patients with inhibitors, but this approach is successful in about 60% of patients. Treatment of acute bleeding in patients with inhibitors is one of the greatest challenges in haemophilia management and is costly. Bypassing agents are the mainstay of treatment in these patients. The aims of this study were to review the most recent publications concerning the costs of inhibitor treatment. We conducted a literature review using PubMed which yielded 63 papers analysing the costs of inhibitor management of which 12 were suitable for our study. Four... (More)
One important complication of patients with severe haemophilia A is the formation of inhibitory antibodies to factor VIII (FVIII). Immune tolerance induction (ITI) is the treatment of choice for patients with inhibitors, but this approach is successful in about 60% of patients. Treatment of acute bleeding in patients with inhibitors is one of the greatest challenges in haemophilia management and is costly. Bypassing agents are the mainstay of treatment in these patients. The aims of this study were to review the most recent publications concerning the costs of inhibitor treatment. We conducted a literature review using PubMed which yielded 63 papers analysing the costs of inhibitor management of which 12 were suitable for our study. Four of eight studies supported the use of activated prothrombin complex concentrate (aPCC) with lower costs, but the remaining four studies showed that recombinant factor VIIa (rFVIIa) had a lower average treatment cost. Of four ITI studies, two supported lifelong cost-effectiveness of ITI vs. bypassing agents and the remaining two papers showed a high cost of inhibitor treatment. Dosages, time between onset of bleeding and treatment, patient characteristics and the price of drugs are some of the important issues that should be considered for further studies. (Less)
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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
haemophilia, costs, inhibitors, clinical studies, bypassing agents
in
European Journal of Haematology
volume
94
pages
30 - 37
publisher
Wiley-Blackwell
external identifiers
  • wos:000347371100007
  • scopus:84920274917
ISSN
1600-0609
DOI
10.1111/ejh.12499
language
English
LU publication?
yes
id
90157bfe-6747-4631-8b30-1d6d6745b2c7 (old id 5076005)
date added to LUP
2015-03-02 07:08:05
date last changed
2017-01-01 03:15:02
@article{90157bfe-6747-4631-8b30-1d6d6745b2c7,
  abstract     = {One important complication of patients with severe haemophilia A is the formation of inhibitory antibodies to factor VIII (FVIII). Immune tolerance induction (ITI) is the treatment of choice for patients with inhibitors, but this approach is successful in about 60% of patients. Treatment of acute bleeding in patients with inhibitors is one of the greatest challenges in haemophilia management and is costly. Bypassing agents are the mainstay of treatment in these patients. The aims of this study were to review the most recent publications concerning the costs of inhibitor treatment. We conducted a literature review using PubMed which yielded 63 papers analysing the costs of inhibitor management of which 12 were suitable for our study. Four of eight studies supported the use of activated prothrombin complex concentrate (aPCC) with lower costs, but the remaining four studies showed that recombinant factor VIIa (rFVIIa) had a lower average treatment cost. Of four ITI studies, two supported lifelong cost-effectiveness of ITI vs. bypassing agents and the remaining two papers showed a high cost of inhibitor treatment. Dosages, time between onset of bleeding and treatment, patient characteristics and the price of drugs are some of the important issues that should be considered for further studies.},
  author       = {Baghaipour, Mohammad Reza and Steen Carlsson, Katarina},
  issn         = {1600-0609},
  keyword      = {haemophilia,costs,inhibitors,clinical studies,bypassing agents},
  language     = {eng},
  pages        = {30--37},
  publisher    = {Wiley-Blackwell},
  series       = {European Journal of Haematology},
  title        = {Strategies for inhibitor treatment and costs in the short and long term: a critical evaluation of recent clinical studies},
  url          = {http://dx.doi.org/10.1111/ejh.12499},
  volume       = {94},
  year         = {2015},
}