Prophylactic treatment for severe haemophilia: comparison of an intermediate-dose to a high-dose regimen
(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:
https://lup.lub.lu.se/record/892518
- author
- Fischer, K ; Astermark, Jan LU ; Van der Bom, JG ; Ljung, R ; Berntorp, Erik LU ; Grobbee, DE and Van den Berg, HM
- organization
- publishing date
- 2002
- 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}}, }