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Prophylactic Treatment of Children with Hemophilia in Sweden

Ljung, Rolf LU orcid (2024) In Seminars in Thrombosis and Hemostasis 50(05). p.714-719
Abstract

Hemophilia A/B are caused by deficiency or lack of coagulation factors VIII (FVIII) or factor IX (FIX), respectively, in plasma. A person with hemophilia develops bleeding in the joints and muscles at an early age, which, if left untreated, leads to early arthropathy. Preventive treatment can be achieved by regular (prophylactic) administration of FVIII/FIX. In 1958, this was implemented on a small scale in Sweden with FVIII in patients with severe hemophilia A, and in those with hemophilia B in 1972 when FIX became available. However, there were problems with human immunodeficiency virus and hepatitis infection from contaminated blood products. In the 1990s, recombinant FVIII and FIX concentrates were introduced. The major remaining... (More)

Hemophilia A/B are caused by deficiency or lack of coagulation factors VIII (FVIII) or factor IX (FIX), respectively, in plasma. A person with hemophilia develops bleeding in the joints and muscles at an early age, which, if left untreated, leads to early arthropathy. Preventive treatment can be achieved by regular (prophylactic) administration of FVIII/FIX. In 1958, this was implemented on a small scale in Sweden with FVIII in patients with severe hemophilia A, and in those with hemophilia B in 1972 when FIX became available. However, there were problems with human immunodeficiency virus and hepatitis infection from contaminated blood products. In the 1990s, recombinant FVIII and FIX concentrates were introduced. The major remaining problems then were the development of inhibitors, and the need for a venous route for the injections in very young children. High-titer inhibitors were treated by immune tolerance induction according to a modified model of the original Bonn high-dose protocol. A central venous line, i.e., Port-A-Cath, has enabled early prophylaxis in many children with poor venous access and has enabled the early start of home treatment with adequate injection frequency. Scoring systems for X-rays, magnetic resonance imaging, and function of joints were developed early in Sweden and have been widely disseminated worldwide, partly with modifications. Extended half-life products with half-life increased three to five times have been developed, which can provide superior bleed protection when dosed once-weekly and can maintain therapeutic trough levels when administered less frequently. The ultimate prophylaxis therapy in the future may be gene therapy.

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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Seminars in Thrombosis and Hemostasis
volume
50
issue
05
pages
6 pages
publisher
Georg Thieme Verlag
external identifiers
  • scopus:85188545637
  • pmid:38499191
ISSN
1098-9064
DOI
10.1055/s-0044-1782518
language
English
LU publication?
yes
id
298760a2-5fb1-4cc0-ac58-1677057b9c20
date added to LUP
2024-03-21 14:35:35
date last changed
2025-07-05 08:07:57
@article{298760a2-5fb1-4cc0-ac58-1677057b9c20,
  abstract     = {{<p>Hemophilia A/B are caused by deficiency or lack of coagulation factors VIII (FVIII) or factor IX (FIX), respectively, in plasma. A person with hemophilia develops bleeding in the joints and muscles at an early age, which, if left untreated, leads to early arthropathy. Preventive treatment can be achieved by regular (prophylactic) administration of FVIII/FIX. In 1958, this was implemented on a small scale in Sweden with FVIII in patients with severe hemophilia A, and in those with hemophilia B in 1972 when FIX became available. However, there were problems with human immunodeficiency virus and hepatitis infection from contaminated blood products. In the 1990s, recombinant FVIII and FIX concentrates were introduced. The major remaining problems then were the development of inhibitors, and the need for a venous route for the injections in very young children. High-titer inhibitors were treated by immune tolerance induction according to a modified model of the original Bonn high-dose protocol. A central venous line, i.e., Port-A-Cath, has enabled early prophylaxis in many children with poor venous access and has enabled the early start of home treatment with adequate injection frequency. Scoring systems for X-rays, magnetic resonance imaging, and function of joints were developed early in Sweden and have been widely disseminated worldwide, partly with modifications. Extended half-life products with half-life increased three to five times have been developed, which can provide superior bleed protection when dosed once-weekly and can maintain therapeutic trough levels when administered less frequently. The ultimate prophylaxis therapy in the future may be gene therapy.</p>}},
  author       = {{Ljung, Rolf}},
  issn         = {{1098-9064}},
  language     = {{eng}},
  month        = {{03}},
  number       = {{05}},
  pages        = {{714--719}},
  publisher    = {{Georg Thieme Verlag}},
  series       = {{Seminars in Thrombosis and Hemostasis}},
  title        = {{Prophylactic Treatment of Children with Hemophilia in Sweden}},
  url          = {{http://dx.doi.org/10.1055/s-0044-1782518}},
  doi          = {{10.1055/s-0044-1782518}},
  volume       = {{50}},
  year         = {{2024}},
}