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Intermediate- dose versus high-dose prophylaxis for severe hemophilia: comparing outcome and costs since the 1970s

Fischer, Kathelijn ; Steen Carlsson, Katarina LU orcid ; Petrini, Pia ; Holmstrom, Margareta ; Ljung, Rolf LU orcid ; van den Berg, H. Marijke and Berntorp, Erik LU (2013) In Blood 122(7). p.1129-1136
Abstract
Prophylactic treatment in severe hemophilia is very effective but is limited by cost issues. The implementation of 2 different prophylactic regimens in The Netherlands and Sweden since the 1970s may be considered a natural experiment. We compared the costs and outcomes of Dutch intermediate-and Swedish high-dose prophylactic regimens for patients with severe hemophilia (factor VIII/IX < 1 IU/dL) born between 1970 and 1994, using prospective standardized outcome assessment and retrospective collection of cost data. Seventy-eight Dutch and 50 Swedish patients, median age 24 years (range, 14-37 years), were included. Intermediate-dose prophylaxis used less factor concentrate (median: Netherlands, 2100 IU/kg per year [interquartile range... (More)
Prophylactic treatment in severe hemophilia is very effective but is limited by cost issues. The implementation of 2 different prophylactic regimens in The Netherlands and Sweden since the 1970s may be considered a natural experiment. We compared the costs and outcomes of Dutch intermediate-and Swedish high-dose prophylactic regimens for patients with severe hemophilia (factor VIII/IX < 1 IU/dL) born between 1970 and 1994, using prospective standardized outcome assessment and retrospective collection of cost data. Seventy-eight Dutch and 50 Swedish patients, median age 24 years (range, 14-37 years), were included. Intermediate-dose prophylaxis used less factor concentrate (median: Netherlands, 2100 IU/kg per year [interquartile range (IQR), 1400-2900 IU/kg per year] vs Sweden, 4000 IU/kg per year [IQR, 3000-4900 IU/kg per year]); (P <.01). Clinical outcome was slightly inferior for the intermediate-dose regimen (P <.01) for 5-year bleeding (median, 1.3 [IQR, 0.8-2.7] vs 0 [IQR, 0.0-2.0] joint bleeds/y) and joint health (Haemophilia Joint Health Score > 10 of 144 points in 46% vs 11% of participants), although social participation and quality of life were similar. Annual total costs were 66% higher for high-dose prophylaxis (mean, 180 [95% confidence interval, 163 -196] 3 US$ 1000 for Dutch vs 298 [95% confidence interval, 271-325]) x US$ 1000 for Swedish patients; (P <.01). At group level, the incremental benefits of high-dose prophylaxis appear limited. At the patient level, prophylaxis should be tailored individually, and many patients may do well receiving lower doses of concentrate without compromising safety. (Less)
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author
; ; ; ; ; and
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Blood
volume
122
issue
7
pages
1129 - 1136
publisher
American Society of Hematology
external identifiers
  • wos:000323392900012
  • scopus:84887192427
  • pmid:23777770
ISSN
1528-0020
DOI
10.1182/blood-2012-12-470898
language
English
LU publication?
yes
id
8b2667ca-63c0-4efe-a387-8a3a7bd18db2 (old id 4063448)
date added to LUP
2016-04-01 09:52:16
date last changed
2023-10-25 18:39:33
@article{8b2667ca-63c0-4efe-a387-8a3a7bd18db2,
  abstract     = {{Prophylactic treatment in severe hemophilia is very effective but is limited by cost issues. The implementation of 2 different prophylactic regimens in The Netherlands and Sweden since the 1970s may be considered a natural experiment. We compared the costs and outcomes of Dutch intermediate-and Swedish high-dose prophylactic regimens for patients with severe hemophilia (factor VIII/IX &lt; 1 IU/dL) born between 1970 and 1994, using prospective standardized outcome assessment and retrospective collection of cost data. Seventy-eight Dutch and 50 Swedish patients, median age 24 years (range, 14-37 years), were included. Intermediate-dose prophylaxis used less factor concentrate (median: Netherlands, 2100 IU/kg per year [interquartile range (IQR), 1400-2900 IU/kg per year] vs Sweden, 4000 IU/kg per year [IQR, 3000-4900 IU/kg per year]); (P &lt;.01). Clinical outcome was slightly inferior for the intermediate-dose regimen (P &lt;.01) for 5-year bleeding (median, 1.3 [IQR, 0.8-2.7] vs 0 [IQR, 0.0-2.0] joint bleeds/y) and joint health (Haemophilia Joint Health Score &gt; 10 of 144 points in 46% vs 11% of participants), although social participation and quality of life were similar. Annual total costs were 66% higher for high-dose prophylaxis (mean, 180 [95% confidence interval, 163 -196] 3 US$ 1000 for Dutch vs 298 [95% confidence interval, 271-325]) x US$ 1000 for Swedish patients; (P &lt;.01). At group level, the incremental benefits of high-dose prophylaxis appear limited. At the patient level, prophylaxis should be tailored individually, and many patients may do well receiving lower doses of concentrate without compromising safety.}},
  author       = {{Fischer, Kathelijn and Steen Carlsson, Katarina and Petrini, Pia and Holmstrom, Margareta and Ljung, Rolf and van den Berg, H. Marijke and Berntorp, Erik}},
  issn         = {{1528-0020}},
  language     = {{eng}},
  number       = {{7}},
  pages        = {{1129--1136}},
  publisher    = {{American Society of Hematology}},
  series       = {{Blood}},
  title        = {{Intermediate- dose versus high-dose prophylaxis for severe hemophilia: comparing outcome and costs since the 1970s}},
  url          = {{http://dx.doi.org/10.1182/blood-2012-12-470898}},
  doi          = {{10.1182/blood-2012-12-470898}},
  volume       = {{122}},
  year         = {{2013}},
}