Intermediate- dose versus high-dose prophylaxis for severe hemophilia: comparing outcome and costs since the 1970s
(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)
Please use this url to cite or link to this publication:
https://lup.lub.lu.se/record/4063448
- author
- Fischer, Kathelijn ; Steen Carlsson, Katarina LU ; Petrini, Pia ; Holmstrom, Margareta ; Ljung, Rolf LU ; van den Berg, H. Marijke and Berntorp, Erik LU
- organization
- publishing date
- 2013
- 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 < 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.}}, 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}}, }