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Controlled, cross-sectional MRI evaluation of joint status in severe haemophilia A patients treated with prophylaxis vs. on demand

Oldenburg, J.; Zimmermann, R.; Katsarou, O.; Theodossiades, G.; Zanon, E.; Niemann, B.; Kellermann, E. and Lundin, Björn LU (2015) In Haemophilia 21(2). p.171-179
Abstract
In patients with haemophilia A, factor VIII (FVIII) prophylaxis reduces bleeding frequency and joint damage compared with on-demand therapy. To assess the effect of prophylaxis initiation age, magnetic resonance imaging (MRI) was used to evaluate bone and cartilage damage in patients with severe haemophilia A. In this cross-sectional, multinational investigation, patients aged 12-35years were assigned to 1 of 5 groups: primary prophylaxis started at age <2years (group 1); secondary prophylaxis started at age 2 to <6years (group 2), 6 to <12years (group 3), or 12-18years (group 4); or on-demand treatment (group 5). Joint status at ankles and knees was assessed using Compatible Additive MRI scoring (maximum and mean ankle; maximum... (More)
In patients with haemophilia A, factor VIII (FVIII) prophylaxis reduces bleeding frequency and joint damage compared with on-demand therapy. To assess the effect of prophylaxis initiation age, magnetic resonance imaging (MRI) was used to evaluate bone and cartilage damage in patients with severe haemophilia A. In this cross-sectional, multinational investigation, patients aged 12-35years were assigned to 1 of 5 groups: primary prophylaxis started at age <2years (group 1); secondary prophylaxis started at age 2 to <6years (group 2), 6 to <12years (group 3), or 12-18years (group 4); or on-demand treatment (group 5). Joint status at ankles and knees was assessed using Compatible Additive MRI scoring (maximum and mean ankle; maximum and mean of all 4 joints) and Gilbert scores in the per-protocol population (n=118). All prophylaxis groups had better MRI joint scores than the on-demand group. MRI scores generally increased with current patient age and later start of prophylaxis. Ankles were the most affected joints. In group 1 patients currently aged 27-35years, the median of maximum ankle scores was 0.0; corresponding values in groups 4 and 5 were 17.0 and 18.0, respectively [medians of mean index joint scores: 0.0 (group 1), 8.1 (group 2) and 13.8 (group 4)]. Gilbert scores revealed outcomes less pronounced than MRI scores. MRI scores identified pathologic joint status with high sensitivity. Prophylaxis groups had lower annualized joint bleeds and MRI scores vs. the on-demand group. Primary prophylaxis demonstrated protective effects against joint deterioration compared with secondary prophylaxis. (Less)
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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
cross-sectional study, haemophilia, joint status, magnetic resonance, imaging, on-demand treatment, prophylaxis
in
Haemophilia
volume
21
issue
2
pages
171 - 179
publisher
Federation of European Neuroscience Societies and Blackwell Publishing Ltd
external identifiers
  • wos:000350065000014
  • scopus:84923106615
ISSN
1351-8216
DOI
10.1111/hae.12539
language
English
LU publication?
yes
id
163d3b85-3660-4ce2-a988-814ae424fa55 (old id 5305141)
date added to LUP
2015-05-04 08:40:58
date last changed
2017-11-12 03:12:51
@article{163d3b85-3660-4ce2-a988-814ae424fa55,
  abstract     = {In patients with haemophilia A, factor VIII (FVIII) prophylaxis reduces bleeding frequency and joint damage compared with on-demand therapy. To assess the effect of prophylaxis initiation age, magnetic resonance imaging (MRI) was used to evaluate bone and cartilage damage in patients with severe haemophilia A. In this cross-sectional, multinational investigation, patients aged 12-35years were assigned to 1 of 5 groups: primary prophylaxis started at age &lt;2years (group 1); secondary prophylaxis started at age 2 to &lt;6years (group 2), 6 to &lt;12years (group 3), or 12-18years (group 4); or on-demand treatment (group 5). Joint status at ankles and knees was assessed using Compatible Additive MRI scoring (maximum and mean ankle; maximum and mean of all 4 joints) and Gilbert scores in the per-protocol population (n=118). All prophylaxis groups had better MRI joint scores than the on-demand group. MRI scores generally increased with current patient age and later start of prophylaxis. Ankles were the most affected joints. In group 1 patients currently aged 27-35years, the median of maximum ankle scores was 0.0; corresponding values in groups 4 and 5 were 17.0 and 18.0, respectively [medians of mean index joint scores: 0.0 (group 1), 8.1 (group 2) and 13.8 (group 4)]. Gilbert scores revealed outcomes less pronounced than MRI scores. MRI scores identified pathologic joint status with high sensitivity. Prophylaxis groups had lower annualized joint bleeds and MRI scores vs. the on-demand group. Primary prophylaxis demonstrated protective effects against joint deterioration compared with secondary prophylaxis.},
  author       = {Oldenburg, J. and Zimmermann, R. and Katsarou, O. and Theodossiades, G. and Zanon, E. and Niemann, B. and Kellermann, E. and Lundin, Björn},
  issn         = {1351-8216},
  keyword      = {cross-sectional study,haemophilia,joint status,magnetic resonance,imaging,on-demand treatment,prophylaxis},
  language     = {eng},
  number       = {2},
  pages        = {171--179},
  publisher    = {Federation of European Neuroscience Societies and Blackwell Publishing Ltd},
  series       = {Haemophilia},
  title        = {Controlled, cross-sectional MRI evaluation of joint status in severe haemophilia A patients treated with prophylaxis vs. on demand},
  url          = {http://dx.doi.org/10.1111/hae.12539},
  volume       = {21},
  year         = {2015},
}