Advanced

Prophylactic treatment for severe haemophilia: comparison of an intermediate-dose to a high-dose regimen

Fischer, K; Astermark, Jan LU ; Van der Bom, JG; Ljung, R; Berntorp, Erik LU ; Grobbee, DE and Van den Berg, HM (2002) In Haemophilia 8(6). p.753-760
Abstract
A multicentre study was performed in Sweden and the Netherlands, comparing effects of two prophylactic regimens in 128 patients with severe haemophilia, born 1970-90. 42 Swedish patients (high-dose prophylaxis), were compared with 86 Dutch patients (intermediate-dose prophylaxis). Patients were evaluated at the date of their last radiological score according to Pettersson. Annual clotting factor consumption and bleeding frequency were registered for a period of three years before evaluation. Patients in the high-dose group were younger at evaluation (median 15.2 vs. 17.9 years), started prophylaxis earlier (median 2 vs. 5 years), and used 2.19 times more clotting factor kg(-1) year(-1). Patients treated with high-dose prophylaxis had fewer... (More)
A multicentre study was performed in Sweden and the Netherlands, comparing effects of two prophylactic regimens in 128 patients with severe haemophilia, born 1970-90. 42 Swedish patients (high-dose prophylaxis), were compared with 86 Dutch patients (intermediate-dose prophylaxis). Patients were evaluated at the date of their last radiological score according to Pettersson. Annual clotting factor consumption and bleeding frequency were registered for a period of three years before evaluation. Patients in the high-dose group were younger at evaluation (median 15.2 vs. 17.9 years), started prophylaxis earlier (median 2 vs. 5 years), and used 2.19 times more clotting factor kg(-1) year(-1). Patients treated with high-dose prophylaxis had fewer joint bleeds (median 0.3 year(-1) vs. 3.3 year(-1)) and the proportion of patients without arthropathy as measured by the Pettersson score was higher (69% vs. 32%), however, the age-adjusted difference in scores (median 0 points vs. 4 points) was small and at present not statistically significant. Clinical scores and quality of life were similar. These findings suggest that, compared with intermediate-dose prophylaxis, high-dose prophylaxis significantly increases treatment costs and reduces joint bleeds over a period of 3 years, but only slightly reduces arthropathy after 17 years of follow-up. (Less)
Please use this url to cite or link to this publication:
author
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
Pettersson, haemarthrosis, clotting factor consumption, arthropathy, prophylaxis, score
in
Haemophilia
volume
8
issue
6
pages
753 - 760
publisher
Federation of European Neuroscience Societies and Blackwell Publishing Ltd
external identifiers
  • wos:000178964200004
  • pmid:12410643
  • scopus:0036827020
ISSN
1351-8216
DOI
10.1046/j.1365-2516.2002.00694.x
language
English
LU publication?
yes
id
d5fd33d7-826a-4f63-879d-0c10f78b0535 (old id 892518)
date added to LUP
2008-01-18 16:08:46
date last changed
2017-11-12 03:30:17
@article{d5fd33d7-826a-4f63-879d-0c10f78b0535,
  abstract     = {A multicentre study was performed in Sweden and the Netherlands, comparing effects of two prophylactic regimens in 128 patients with severe haemophilia, born 1970-90. 42 Swedish patients (high-dose prophylaxis), were compared with 86 Dutch patients (intermediate-dose prophylaxis). Patients were evaluated at the date of their last radiological score according to Pettersson. Annual clotting factor consumption and bleeding frequency were registered for a period of three years before evaluation. Patients in the high-dose group were younger at evaluation (median 15.2 vs. 17.9 years), started prophylaxis earlier (median 2 vs. 5 years), and used 2.19 times more clotting factor kg(-1) year(-1). Patients treated with high-dose prophylaxis had fewer joint bleeds (median 0.3 year(-1) vs. 3.3 year(-1)) and the proportion of patients without arthropathy as measured by the Pettersson score was higher (69% vs. 32%), however, the age-adjusted difference in scores (median 0 points vs. 4 points) was small and at present not statistically significant. Clinical scores and quality of life were similar. These findings suggest that, compared with intermediate-dose prophylaxis, high-dose prophylaxis significantly increases treatment costs and reduces joint bleeds over a period of 3 years, but only slightly reduces arthropathy after 17 years of follow-up.},
  author       = {Fischer, K and Astermark, Jan and Van der Bom, JG and Ljung, R and Berntorp, Erik and Grobbee, DE and Van den Berg, HM},
  issn         = {1351-8216},
  keyword      = {Pettersson,haemarthrosis,clotting factor consumption,arthropathy,prophylaxis,score},
  language     = {eng},
  number       = {6},
  pages        = {753--760},
  publisher    = {Federation of European Neuroscience Societies and Blackwell Publishing Ltd},
  series       = {Haemophilia},
  title        = {Prophylactic treatment for severe haemophilia: comparison of an intermediate-dose to a high-dose regimen},
  url          = {http://dx.doi.org/10.1046/j.1365-2516.2002.00694.x},
  volume       = {8},
  year         = {2002},
}