Long-term joint outcomes in adolescents with moderate or severe haemophilia A
© 2022 The Authors. Haemophilia published by John Wiley & Sons Ltd..
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.
| Medienart: |
E-Artikel |
|---|
| Erscheinungsjahr: |
2022 |
|---|---|
| Erschienen: |
2022 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:28 |
|---|---|
| Enthalten in: |
Haemophilia : the official journal of the World Federation of Hemophilia - 28(2022), 6 vom: 08. Nov., Seite 1054-1061 |
| Sprache: |
Englisch |
|---|
| Beteiligte Personen: |
Schmidt, David E [VerfasserIn] |
|---|
| Links: |
|---|
| Themen: |
Adolescent |
|---|
| Anmerkungen: |
Date Completed 15.11.2022 Date Revised 07.01.2023 published: Print-Electronic Citation Status MEDLINE |
|---|
| doi: |
10.1111/hae.14636 |
|---|
| funding: |
|
|---|---|
| Förderinstitution / Projekttitel: |
|
| PPN (Katalog-ID): |
NLM344451054 |
|---|
| LEADER | 01000naa a22002652 4500 | ||
|---|---|---|---|
| 001 | NLM344451054 | ||
| 003 | DE-627 | ||
| 005 | 20231226022814.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 231226s2022 xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.1111/hae.14636 |2 doi | |
| 028 | 5 | 2 | |a pubmed24n1148.xml |
| 035 | |a (DE-627)NLM344451054 | ||
| 035 | |a (NLM)35925557 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rakwb | ||
| 041 | |a eng | ||
| 100 | 1 | |a Schmidt, David E |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Long-term joint outcomes in adolescents with moderate or severe haemophilia A |
| 264 | 1 | |c 2022 | |
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a ƒaComputermedien |b c |2 rdamedia | ||
| 338 | |a ƒa Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Date Completed 15.11.2022 | ||
| 500 | |a Date Revised 07.01.2023 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a © 2022 The Authors. Haemophilia published by John Wiley & Sons Ltd. | ||
| 520 | |a 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 | ||
| 520 | |a AIM: To measure the long-term joint health of adolescents with moderate and severe HA, according to severity and inhibitor status | ||
| 520 | |a 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) | ||
| 520 | |a 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%) | ||
| 520 | |a 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 | ||
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a adolescent | |
| 650 | 4 | |a arthropathy | |
| 650 | 4 | |a haemophilia A | |
| 650 | 4 | |a paediatrics | |
| 650 | 4 | |a ultrasonography | |
| 700 | 1 | |a Michalopoulou, Aikaterini |e verfasserin |4 aut | |
| 700 | 1 | |a Fischer, Kathelijn |e verfasserin |4 aut | |
| 700 | 1 | |a Motwani, Jayashree |e verfasserin |4 aut | |
| 700 | 1 | |a Andersson, Nadine G |e verfasserin |4 aut | |
| 700 | 1 | |a Pergantou, Helen |e verfasserin |4 aut | |
| 700 | 1 | |a Ranta, Susanna |e verfasserin |4 aut | |
| 700 | 0 | |a PedNet Study Group |e verfasserin |4 aut | |
| 773 | 0 | 8 | |i Enthalten in |t Haemophilia : the official journal of the World Federation of Hemophilia |d 1995 |g 28(2022), 6 vom: 08. Nov., Seite 1054-1061 |w (DE-627)NLM093860773 |x 1365-2516 |7 nnns |
| 773 | 1 | 8 | |g volume:28 |g year:2022 |g number:6 |g day:08 |g month:11 |g pages:1054-1061 |
| 856 | 4 | 0 | |u http://dx.doi.org/10.1111/hae.14636 |3 Volltext |
| 912 | |a GBV_USEFLAG_A | ||
| 912 | |a GBV_NLM | ||
| 951 | |a AR | ||
| 952 | |d 28 |j 2022 |e 6 |b 08 |c 11 |h 1054-1061 | ||