Long-term joint outcomes in adolescents with moderate or severe haemophilia A
(2022) In Haemophilia 28(6). p.1054-1061- Abstract
Introduction: Favourable joint outcomes are expected with modern haemophilia A (HA) management. Evaluation of long-term treatment outcomes is hampered by the delay between bleeding episodes during childhood and resulting joint outcomes in adulthood. Aim: To measure the long-term joint health of adolescents with moderate and severe HA, according to severity and inhibitor status. Methods: Pilot cross-sectional study of five European PedNet centres in moderate and severe HA patients aged 10–19 years. Structured assessment of joint status by physical examination (HJHS) and ultrasound (HEAD-US). Results: In total, 141 HA patients were evaluable, 100 without inhibitors (81 severe, 19 moderate HA), and 41 severe HA with current/past... (More)
Introduction: Favourable joint outcomes are expected with modern haemophilia A (HA) management. Evaluation of long-term treatment outcomes is hampered by the delay between bleeding episodes during childhood and resulting joint outcomes in adulthood. Aim: To measure the long-term joint health of adolescents with moderate and severe HA, according to severity and inhibitor status. Methods: Pilot cross-sectional study of five European PedNet centres in moderate and severe HA patients aged 10–19 years. Structured assessment of joint status by physical examination (HJHS) and ultrasound (HEAD-US). Results: In total, 141 HA patients were evaluable, 100 without inhibitors (81 severe, 19 moderate HA), and 41 severe HA with current/past inhibitors. On physical examination, 12/81 (15%) of severe HA without inhibitors, 3/19 (16%) of moderate HA, and 13/41 (32%) of severe HA patients with inhibitors exhibited joint abnormalities. Inhibitor persistence, longer inhibitor duration, and a high peak inhibitor level were associated with impaired joint health. Ultrasound showed joint damage (bone or cartilage) in 13/49 (27%) of severe HA without inhibitors, 1/12 (8%) of moderate HA, and 10/28 (36%) of severe HA patients with inhibitors. A discordant ankle evaluation by ultrasound versus physical examination was present in 53/169 joints (31%). Conclusions: Most adolescents with severe or moderate HA show favourable joint health. Future research with combined ultrasound and/or MRI is needed to better understand joint outcomes in the remaining patients. Patents with inhibitors showed a two-fold increased proportion with joint deterioration. Ultrasound paired with physical examination increases sensitivity for detection of joint damage.
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- author
- Schmidt, David E. ; Michalopoulou, Aikaterini ; Fischer, Kathelijn ; Motwani, Jayashree ; Andersson, Nadine G. LU ; Pergantou, Helen and Ranta, Susanna
- author collaboration
- organization
- publishing date
- 2022
- type
- Contribution to journal
- publication status
- published
- subject
- keywords
- adolescent, arthropathy, haemophilia A, paediatrics, ultrasonography
- in
- Haemophilia
- volume
- 28
- issue
- 6
- pages
- 1054 - 1061
- publisher
- Wiley-Blackwell
- external identifiers
-
- scopus:85135528107
- pmid:35925557
- ISSN
- 1351-8216
- DOI
- 10.1111/hae.14636
- language
- English
- LU publication?
- yes
- id
- c75fdd12-32cb-4272-ab25-3249030a6181
- date added to LUP
- 2022-10-07 14:36:38
- date last changed
- 2024-06-13 20:02:49
@article{c75fdd12-32cb-4272-ab25-3249030a6181,
abstract = {{<p>Introduction: Favourable joint outcomes are expected with modern haemophilia A (HA) management. Evaluation of long-term treatment outcomes is hampered by the delay between bleeding episodes during childhood and resulting joint outcomes in adulthood. Aim: To measure the long-term joint health of adolescents with moderate and severe HA, according to severity and inhibitor status. Methods: Pilot cross-sectional study of five European PedNet centres in moderate and severe HA patients aged 10–19 years. Structured assessment of joint status by physical examination (HJHS) and ultrasound (HEAD-US). Results: In total, 141 HA patients were evaluable, 100 without inhibitors (81 severe, 19 moderate HA), and 41 severe HA with current/past inhibitors. On physical examination, 12/81 (15%) of severe HA without inhibitors, 3/19 (16%) of moderate HA, and 13/41 (32%) of severe HA patients with inhibitors exhibited joint abnormalities. Inhibitor persistence, longer inhibitor duration, and a high peak inhibitor level were associated with impaired joint health. Ultrasound showed joint damage (bone or cartilage) in 13/49 (27%) of severe HA without inhibitors, 1/12 (8%) of moderate HA, and 10/28 (36%) of severe HA patients with inhibitors. A discordant ankle evaluation by ultrasound versus physical examination was present in 53/169 joints (31%). Conclusions: Most adolescents with severe or moderate HA show favourable joint health. Future research with combined ultrasound and/or MRI is needed to better understand joint outcomes in the remaining patients. Patents with inhibitors showed a two-fold increased proportion with joint deterioration. Ultrasound paired with physical examination increases sensitivity for detection of joint damage.</p>}},
author = {{Schmidt, David E. and Michalopoulou, Aikaterini and Fischer, Kathelijn and Motwani, Jayashree and Andersson, Nadine G. and Pergantou, Helen and Ranta, Susanna}},
issn = {{1351-8216}},
keywords = {{adolescent; arthropathy; haemophilia A; paediatrics; ultrasonography}},
language = {{eng}},
number = {{6}},
pages = {{1054--1061}},
publisher = {{Wiley-Blackwell}},
series = {{Haemophilia}},
title = {{Long-term joint outcomes in adolescents with moderate or severe haemophilia A}},
url = {{http://dx.doi.org/10.1111/hae.14636}},
doi = {{10.1111/hae.14636}},
volume = {{28}},
year = {{2022}},
}