title: Optimising musculoskeletal care for patients with haemophilia.
abstract: Despite recent improvements in the quality of care and treatment outcomes for haemophilia, joint disease remains a major concern for patients with and without inhibitors. Most bleeding episodes occur in the musculoskeletal system, and recurrent bleeding may result in progressive joint damage, leading to haemophilic arthropathy. Consequently, early identification and management of musculoskeletal bleeding episodes are important to prevent crippling deformities and dysfunction. Management strategies should aim at optimising joint function by reducing the frequency of, and preventing, joint bleeds. Although prophylactic factor replacement is proven to be effective in reducing bleeding frequency into joints and preserving musculoskeletal function in patients without inhibitors, the role for prophylaxis (with bypassing agents) in patients with inhibitors remains unclear. The available bypassing agents, activated prothrombin complex concentrate and recombinant activated factor VII (rFVIIa), are currently the standard of care for acute bleeding episodes in patients with high-titre inhibitors. These agents also prevent bleeding during elective orthopaedic surgery (EOS) in this patient population. This review discusses published data and uses illustrative cases to describe effective strategies for assessing joint health and maintaining optimal musculoskeletal care, focusing on the use of rFVIIa for haemostatic control in haemarthroses and when EOS is required in patients with inhibitors.
authors.affiliation: Division of Haematology/Oncology and Child Health Evaluative Sciences, Research Institute, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada.
authors.forename: Manuel
authors.initials: M
authors.lastname: Carcao
authors.affiliation: Department of Rehabilitation, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada.
authors.forename: Pamela
authors.initials: P
authors.lastname: Hilliard
authors.affiliation: Gulf States Hemophilia and Thrombophilia Center, Department of Hematology, University of Texas Health Science Center, Houston, TX, USA.
authors.forename: Miguel A
authors.initials: MA
authors.lastname: Escobar
authors.affiliation: Ortho Trauma Unit, Emergency Department, IRCCS Cà Granda Foundation, Maggiore Hospital, Milan, Italy.
authors.forename: Luigi
authors.initials: L
authors.lastname: Solimeno
authors.affiliation: Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service, Johannesburg, South Africa.
authors.forename: Johnny
authors.initials: J
authors.lastname: Mahlangu
authors.affiliation: Hemophilia and Thrombosis Center 'A. Bianchi Bonomi', IRCCS Cà Granda Foundation, Maggiore Hospital, Milan, Italy.
authors.forename: Elena
authors.initials: E
authors.lastname: Santagostino
mesh_terms: D002648:Child; D006467:Hemophilia A / Q000150:complications / Q000503:physiopathology*; D006801:Humans; D007592:Joint Diseases / Q000209:etiology / Q000628:therapy*; D008297:Male
keywords: bypassing agents; elective orthopaedic surgery; haemophilia; inhibitors; joint; multidisciplinary; musculoskeletal; recombinant activated factor VII