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Plasma-derived versus recombinant factor concentrates in PUPs : A never ending debate?

Berntorp, Erik LU (2017) In Hamostaseologie 37(1). p.53-57
Abstract

Inhibitor development in haemophilia is a serious complication to treatment with factor concentrates. Since the advent of more pure products, especially developed using recombinant DNA technology, some studies have shown an increased incidence of inhibitors in previously untreated patients (PUPs) receiving recombinant products whereas plasmaderived concentrates sometimes have been claimed to have a protective role, probably due to the content of von Willebrand factor (VWF). In fact, experiments indicate that the VWF may block uptake of factor VIII into macrophages for further processing to the immune system. Also, a competition between VWF and inhibitor binding to the C2 domain of factor VIII has been suggested. Recently, large cohort... (More)

Inhibitor development in haemophilia is a serious complication to treatment with factor concentrates. Since the advent of more pure products, especially developed using recombinant DNA technology, some studies have shown an increased incidence of inhibitors in previously untreated patients (PUPs) receiving recombinant products whereas plasmaderived concentrates sometimes have been claimed to have a protective role, probably due to the content of von Willebrand factor (VWF). In fact, experiments indicate that the VWF may block uptake of factor VIII into macrophages for further processing to the immune system. Also, a competition between VWF and inhibitor binding to the C2 domain of factor VIII has been suggested. Recently, large cohort and surveillance studies have created a vigorous debate about the role of product class for inhibitor development as results have been conflicting. The only randomised prospective study, the SIPPET study, was published in 2016, and substantiated previous reports claiming that plasma derived concentrates give less inhibitors in patients with severe haemophilia A, previously not exposed to factor VIII. The debate will continue.

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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
Factor concentrate, Haemophilia, Inhibitor, Previously untreated patients
in
Hamostaseologie
volume
37
issue
1
pages
5 pages
publisher
Schattauer
external identifiers
  • scopus:85019409228
  • wos:000394202600007
ISSN
0720-9355
DOI
10.5482/HAMO-16-10-0040
language
English
LU publication?
yes
id
269f951a-6534-488f-b30e-a97febfef1ce
date added to LUP
2017-06-16 13:19:36
date last changed
2018-01-07 12:08:17
@article{269f951a-6534-488f-b30e-a97febfef1ce,
  abstract     = {<p>Inhibitor development in haemophilia is a serious complication to treatment with factor concentrates. Since the advent of more pure products, especially developed using recombinant DNA technology, some studies have shown an increased incidence of inhibitors in previously untreated patients (PUPs) receiving recombinant products whereas plasmaderived concentrates sometimes have been claimed to have a protective role, probably due to the content of von Willebrand factor (VWF). In fact, experiments indicate that the VWF may block uptake of factor VIII into macrophages for further processing to the immune system. Also, a competition between VWF and inhibitor binding to the C2 domain of factor VIII has been suggested. Recently, large cohort and surveillance studies have created a vigorous debate about the role of product class for inhibitor development as results have been conflicting. The only randomised prospective study, the SIPPET study, was published in 2016, and substantiated previous reports claiming that plasma derived concentrates give less inhibitors in patients with severe haemophilia A, previously not exposed to factor VIII. The debate will continue.</p>},
  author       = {Berntorp, Erik},
  issn         = {0720-9355},
  keyword      = {Factor concentrate,Haemophilia,Inhibitor,Previously untreated patients},
  language     = {eng},
  number       = {1},
  pages        = {53--57},
  publisher    = {Schattauer},
  series       = {Hamostaseologie},
  title        = {Plasma-derived versus recombinant factor concentrates in PUPs : A never ending debate?},
  url          = {http://dx.doi.org/10.5482/HAMO-16-10-0040},
  volume       = {37},
  year         = {2017},
}