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Surgery and survival in birth cohorts with severe haemophilia and differences in access to replacement therapy : The Malmö experience

Osooli, M. LU ; Steen Carlsson, K. LU ; Astermark, J. LU and Berntorp, E. LU (2017) In Haemophilia 23(5). p.403-408
Abstract

Background: Persons with severe haemophilia require lifelong replacement therapy, prophylaxis, to prevent bleeding. Data describing long-term outcomes of prophylactic treatment are scarce. The aim of this study was to investigate joint surgery and survival among persons with severe haemophilia with special attention to access to prophylaxis in the early years of life. Methods: Eligible participants had severe haemophilia A or B and were treated at the Malmö centre from the 1960s onward. Time from birth until joint surgery was analysed for participants negative for factor inhibitor and alive in 2000. We compared survival among the entire cohort with severe haemophilia treated at the Malmö centre with the general male population of Sweden... (More)

Background: Persons with severe haemophilia require lifelong replacement therapy, prophylaxis, to prevent bleeding. Data describing long-term outcomes of prophylactic treatment are scarce. The aim of this study was to investigate joint surgery and survival among persons with severe haemophilia with special attention to access to prophylaxis in the early years of life. Methods: Eligible participants had severe haemophilia A or B and were treated at the Malmö centre from the 1960s onward. Time from birth until joint surgery was analysed for participants negative for factor inhibitor and alive in 2000. We compared survival among the entire cohort with severe haemophilia treated at the Malmö centre with the general male population of Sweden and a sample of persons with severe haemophilia from the United Kingdom (UK). Results: Overall, 167 participants were included, 106 (63.5%) of whom had complete data on joint surgery. Among those born before 1970, 1970-1979 and ≥1980 approximately 37%, 21% and 0% had their first joint surgery by age 30, respectively. There were no second joint surgeries reported in cohorts born ≥1970. Persons with severe haemophilia and negative for HIV treated in Malmö have attained approximately similar survival to that of the general male population in Sweden and live slightly longer than persons with severe haemophilia from the UK. Discussion and conclusion: Prophylaxis in Sweden, although costly, has markedly improved survival and joint outcomes for persons with severe haemophilia. This study highlights the importance of early start of replacement therapy to prevent or postpone serious joint damage.

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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
Factor VIII/IX deficiency, Haemophilia, Joint surgery, Register-based study, Survival, Treatment outcomes
in
Haemophilia
volume
23
issue
5
pages
403 - 408
publisher
Federation of European Neuroscience Societies and Blackwell Publishing Ltd
external identifiers
  • scopus:85026443791
  • wos:000411824500002
ISSN
1351-8216
DOI
10.1111/hae.13302
language
English
LU publication?
yes
id
6ad55bb4-751d-41a9-8c4b-920cf8c3dade
date added to LUP
2017-08-30 11:47:21
date last changed
2018-01-16 13:24:03
@article{6ad55bb4-751d-41a9-8c4b-920cf8c3dade,
  abstract     = {<p>Background: Persons with severe haemophilia require lifelong replacement therapy, prophylaxis, to prevent bleeding. Data describing long-term outcomes of prophylactic treatment are scarce. The aim of this study was to investigate joint surgery and survival among persons with severe haemophilia with special attention to access to prophylaxis in the early years of life. Methods: Eligible participants had severe haemophilia A or B and were treated at the Malmö centre from the 1960s onward. Time from birth until joint surgery was analysed for participants negative for factor inhibitor and alive in 2000. We compared survival among the entire cohort with severe haemophilia treated at the Malmö centre with the general male population of Sweden and a sample of persons with severe haemophilia from the United Kingdom (UK). Results: Overall, 167 participants were included, 106 (63.5%) of whom had complete data on joint surgery. Among those born before 1970, 1970-1979 and ≥1980 approximately 37%, 21% and 0% had their first joint surgery by age 30, respectively. There were no second joint surgeries reported in cohorts born ≥1970. Persons with severe haemophilia and negative for HIV treated in Malmö have attained approximately similar survival to that of the general male population in Sweden and live slightly longer than persons with severe haemophilia from the UK. Discussion and conclusion: Prophylaxis in Sweden, although costly, has markedly improved survival and joint outcomes for persons with severe haemophilia. This study highlights the importance of early start of replacement therapy to prevent or postpone serious joint damage.</p>},
  author       = {Osooli, M. and Steen Carlsson, K. and Astermark, J. and Berntorp, E.},
  issn         = {1351-8216},
  keyword      = {Factor VIII/IX deficiency,Haemophilia,Joint surgery,Register-based study,Survival,Treatment outcomes},
  language     = {eng},
  number       = {5},
  pages        = {403--408},
  publisher    = {Federation of European Neuroscience Societies and Blackwell Publishing Ltd},
  series       = {Haemophilia},
  title        = {Surgery and survival in birth cohorts with severe haemophilia and differences in access to replacement therapy : The Malmö experience},
  url          = {http://dx.doi.org/10.1111/hae.13302},
  volume       = {23},
  year         = {2017},
}