TY - JOUR
T1 - How to assess, detect, and manage joint involvement in the era of transformational therapies
T2 - Role of point-of-care ultrasound
AU - Minno, Matteo Nicola Dario Di
AU - Martinoli, Carlo
AU - Pasta, Gianluigi
AU - la Corte-Rodriguez, Hortensia De
AU - Samy, Islam
AU - Stephensen, David
AU - Timmer, Merel A
AU - Winburn, Ian
N1 - Funding Information:
Matteo Nicola Dario Di Minno has received research funding from and served as a consultant for Bayer, Pfizer, Novo Nordisk, Roche, Sobi and Takeda. Carlo Martinoli has served as a paid consultant for Pfizer, and has received speaker fees from Canon, Esaote, Novartis, Novo Nordisk, Pfizer, Philips, Sobi, Roche and Takeda. Gianluigi Pasta has received honoraria for attending symposia/congresses and/or for speaking and/or consulting, and/or has received research funding from Bayer, Novo Nordisk, Pfizer, Roche, Sobi and Takeda. Hortensia De la Corte‐Rodriguez has received honoraria for attending symposia/congresses and/or for speaking and/or consulting, and/or has received research funding from Bayer, Novo Nordisk, Pfizer, Roche, Sobi and Takeda. David Stephensen has received research funding from Bayer, CSL Behring, the National Institute for Health Research, Novo Nordisk, Pfizer, Sobi, and Roche; has served as a speaker for Pfizer, Sanofi Genzyme/Sobi, Sanofi and Takeda; and has served as a remunerated consultant for Roche, Sobi and Takeda. Merel Timmer has received research grants from Novo Nordisk, Sobi, and Pfizer. Islam Samy and Ian Winburn are employees of Pfizer Inc and own stock in the company.
Publisher Copyright:
© 2022 John Wiley & Sons Ltd.
PY - 2023/1
Y1 - 2023/1
N2 - BACKGROUND: Patients with haemophilia experience recurring hemarthroses, mainly involving knees, elbows and ankles, which lead to haemophilic arthropathy, the major chronic complication of haemophilia. With new approaches to haemophilia treatment leading to fewer joint bleeds and, in some cases, no bleeding events, assessing whether current outcome assessment tools provide adequate sensitivity and specificity for management and care of patients with haemophilia is needed.METHODS: An overview of current imaging tools for monitoring joint health, novel osteochondral damage and synovial proliferation biomarkers, and the relationship between assessments for functionality and imaging modalities is provided. Usefulness and sensitivity of point-of-care ultrasound (POCUS) to complement other assessments and use of ultrasound to monitor haemophilic arthropathy are also examined.RESULTS: This review provides rationale for haemophilia teams to move beyond traditional outcomes in joint imaging, as well as guidance and evidence on assessment of joint health for potential new treatment modalities, such as gene therapy. The role of POCUS in the existing paradigm for haemophilia care and management along with the use of ultrasound as a complement to other outcome assessment tools are also discussed. Finally, the clinical effects of subclinical bleeding on joint function are described, to motivate screening for synovial proliferation.CONCLUSION: POCUS can facilitate the early detection of joint damage and can monitor disease progression while providing insights into the efficacy of treatment regimens, and should be considered as an essential assessment tool for managing the care of patients with haemophilia.
AB - BACKGROUND: Patients with haemophilia experience recurring hemarthroses, mainly involving knees, elbows and ankles, which lead to haemophilic arthropathy, the major chronic complication of haemophilia. With new approaches to haemophilia treatment leading to fewer joint bleeds and, in some cases, no bleeding events, assessing whether current outcome assessment tools provide adequate sensitivity and specificity for management and care of patients with haemophilia is needed.METHODS: An overview of current imaging tools for monitoring joint health, novel osteochondral damage and synovial proliferation biomarkers, and the relationship between assessments for functionality and imaging modalities is provided. Usefulness and sensitivity of point-of-care ultrasound (POCUS) to complement other assessments and use of ultrasound to monitor haemophilic arthropathy are also examined.RESULTS: This review provides rationale for haemophilia teams to move beyond traditional outcomes in joint imaging, as well as guidance and evidence on assessment of joint health for potential new treatment modalities, such as gene therapy. The role of POCUS in the existing paradigm for haemophilia care and management along with the use of ultrasound as a complement to other outcome assessment tools are also discussed. Finally, the clinical effects of subclinical bleeding on joint function are described, to motivate screening for synovial proliferation.CONCLUSION: POCUS can facilitate the early detection of joint damage and can monitor disease progression while providing insights into the efficacy of treatment regimens, and should be considered as an essential assessment tool for managing the care of patients with haemophilia.
KW - haemarthrosis
KW - haemophilia A
KW - haemorrhage
KW - joint diseases
KW - ultrasound
UR - https://www.scopus.com/pages/publications/85138687761
U2 - 10.1111/hae.14657
DO - 10.1111/hae.14657
M3 - Review article
C2 - 36163646
SN - 1351-8216
VL - 29
SP - 1
EP - 10
JO - Haemophilia
JF - Haemophilia
IS - 1
ER -