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On-demand treatment in persons with severe haemophilia

Trakymiene, Sonata Saulyte and Steen Carlsson, Katarina LU (2014) In European Journal of Haematology 93. p.39-47
Abstract
There are two main modes of replacement therapy for haemophilia patients: either to stop bleeding (on-demand) or regular infusions of clotting factor to prevent bleeds (prophylaxis). Fiftyyr of clinical experience have provided evidence of the superiority of prophylaxis by showing a reduction in bleeds and development of arthropathy. Prophylaxis has been described extensively in terms of efficacy and health-economic aspects; however, on-demand treatment has received less attention. The aim of this study was to critically review the published literature on PubMed and discuss potential gaps of knowledge in on-demand treatment in persons with severe haemophilia without inhibitors by focusing on two key aspects: how on-demand treatment is... (More)
There are two main modes of replacement therapy for haemophilia patients: either to stop bleeding (on-demand) or regular infusions of clotting factor to prevent bleeds (prophylaxis). Fiftyyr of clinical experience have provided evidence of the superiority of prophylaxis by showing a reduction in bleeds and development of arthropathy. Prophylaxis has been described extensively in terms of efficacy and health-economic aspects; however, on-demand treatment has received less attention. The aim of this study was to critically review the published literature on PubMed and discuss potential gaps of knowledge in on-demand treatment in persons with severe haemophilia without inhibitors by focusing on two key aspects: how on-demand treatment is provided and what outcome measures have been reported. We identified 134 papers of which 112 were excluded. Of the remaining 22 papers, 16 were comparative studies between prophylaxis and on-demand treatment and six were descriptions of on-demand treatment. The results showed limited reporting on data related to the key aspects of treatment on-demand. Early studies looked at degrees of joint bleeds and different treatment regimens in finding the optimal dose. However, from the late 1980s, there was almost no research into on-demand therapy except efficacy and safety studies of new rFVIII products and studies to prove superiority of prophylaxis over treatment on-demand. The success of on-demand therapy may depend on several factors, for example time to initial dose after a bleed and duration of treatment. Data on these key factors are limited and highlight the necessity of research to optimise replacement therapy. (Less)
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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
haemophilia, on-demand treatment, prophylaxis, literature review
in
European Journal of Haematology
volume
93
pages
39 - 47
publisher
Wiley-Blackwell
external identifiers
  • wos:000338019900006
  • scopus:84902952509
ISSN
1600-0609
DOI
10.1111/ejh.12373
language
English
LU publication?
yes
id
00805a4d-65fd-4c85-beba-691384bb5d8e (old id 4608867)
date added to LUP
2014-09-01 07:36:55
date last changed
2017-01-01 03:51:10
@article{00805a4d-65fd-4c85-beba-691384bb5d8e,
  abstract     = {There are two main modes of replacement therapy for haemophilia patients: either to stop bleeding (on-demand) or regular infusions of clotting factor to prevent bleeds (prophylaxis). Fiftyyr of clinical experience have provided evidence of the superiority of prophylaxis by showing a reduction in bleeds and development of arthropathy. Prophylaxis has been described extensively in terms of efficacy and health-economic aspects; however, on-demand treatment has received less attention. The aim of this study was to critically review the published literature on PubMed and discuss potential gaps of knowledge in on-demand treatment in persons with severe haemophilia without inhibitors by focusing on two key aspects: how on-demand treatment is provided and what outcome measures have been reported. We identified 134 papers of which 112 were excluded. Of the remaining 22 papers, 16 were comparative studies between prophylaxis and on-demand treatment and six were descriptions of on-demand treatment. The results showed limited reporting on data related to the key aspects of treatment on-demand. Early studies looked at degrees of joint bleeds and different treatment regimens in finding the optimal dose. However, from the late 1980s, there was almost no research into on-demand therapy except efficacy and safety studies of new rFVIII products and studies to prove superiority of prophylaxis over treatment on-demand. The success of on-demand therapy may depend on several factors, for example time to initial dose after a bleed and duration of treatment. Data on these key factors are limited and highlight the necessity of research to optimise replacement therapy.},
  author       = {Trakymiene, Sonata Saulyte and Steen Carlsson, Katarina},
  issn         = {1600-0609},
  keyword      = {haemophilia,on-demand treatment,prophylaxis,literature review},
  language     = {eng},
  pages        = {39--47},
  publisher    = {Wiley-Blackwell},
  series       = {European Journal of Haematology},
  title        = {On-demand treatment in persons with severe haemophilia},
  url          = {http://dx.doi.org/10.1111/ejh.12373},
  volume       = {93},
  year         = {2014},
}