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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)
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author
; ; ; ; ; and
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
Wiley-Blackwell
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
additional info
The information about affiliations in this record was updated in December 2015. The record was previously connected to the following departments: Clinical Coagulation Research Unit (013242510), Emergency medicine/Medicine/Surgery (013240200)
id
d5fd33d7-826a-4f63-879d-0c10f78b0535 (old id 892518)
date added to LUP
2016-04-01 12:25:55
date last changed
2022-07-22 20:29:00
@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}},
  keywords     = {{Pettersson; haemarthrosis; clotting factor consumption; arthropathy; prophylaxis; score}},
  language     = {{eng}},
  number       = {{6}},
  pages        = {{753--760}},
  publisher    = {{Wiley-Blackwell}},
  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}},
  doi          = {{10.1046/j.1365-2516.2002.00694.x}},
  volume       = {{8}},
  year         = {{2002}},
}