Journal ArticleDOI
Haemophilia care in Europe: the ESCHQoL study.
W. Schramm,Alessandro Gringeri,Rolf Ljung,Karin Berger,Alexander Crispin,Monika Bullinger,Paul L. F. Giangrande,S. von Mackensen,Lorenzo G. Mantovani,László Nemes,Margit Serban +10 more
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TLDR
Statistical analysis revealed that residence in areas with low factor consumption/availability is the most prominent risk factor for joint disease and access of European patients with haemophilia to optimal care with safe factor VIII concentrates is limited and depends on the region of residence.Abstract:
The aim of this study was to determine the clinical conditions of patients with haemophilia within Europe as recommended by the European Commission. In this multicentre, cross-sectional, ambispective study, conducted within 21 European countries patients' clinical data were collected, amongst others haemophilia type, severity, treatment pattern, use of factor products, bleeding, orthopaedic joint scores and infections. A total of 1400 patients, 84.3% with haemophilia A and 15.7% with haemophilia B were enrolled by 42 centres between 2004 and 2006. Thereof, 417 were children (30.0%) and 983 were adults (70.0%). About 70% of patients had severe factor deficiency ( 5 IU; region 2: 2-5 IU; region 3: <2 IU. Paediatric and adult patients in region 3 had median numbers of three and eight joint bleeds, respectively, with worse joint scores compared to region 1 with zero and one bleed. Prophylactic therapy was used in only 31.3% children and 8.9% adults with severe haemophilia in region 3 compared to 93.7% and 54.1%, respectively, in region 1. Statistical analysis revealed that residence in areas with low factor consumption/availability is the most prominent risk factor for joint disease. Access of European patients with haemophilia to optimal care with safe factor VIII concentrates is limited and depends on the region of residence.read more
Citations
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Journal ArticleDOI
Advances and challenges in hemophilic arthropathy
TL;DR: Current imaging techniques and the development of high resolution musculoskeletal ultrasound with power Doppler to afford point-of-care diagnosis and management, the potential utility of diagnostic biomarkers, and the current knowledge of the pathobiology of hemophilic arthropathy are described.
Journal ArticleDOI
Vascular remodeling underlies rebleeding in hemophilic arthropathy.
Vikas Bhat,Vikas Bhat,Merissa Olmer,Shweta Joshi,Donald L. Durden,Thomas J. Cramer,Richard F. W. Barnes,Scott T. Ball,Tudor H. Hughes,Mauricio Silva,James V. Luck,Randy E. Moore,Laurent O. Mosnier,Annette von Drygalski,Annette von Drygalski +14 more
TL;DR: Observations support the hypothesis that vascular remodeling contributes significantly to bleed propagation and development of hemophilic arthropathy and the development of molecular targets for angiogenesis inhibition may be considered in this disease.
Journal ArticleDOI
Haemophilia patients' unmet needs and their expectations of the new extended half-life factor concentrates.
S. von Mackensen,Werner Kalnins,J. Krucker,J. Weiss,Wolfgang Miesbach,Manuela Albisetti,Ingrid Pabinger,Johannes Oldenburg +7 more
TL;DR: National Member Organisations (NMO) of persons with haemophilia from the DACH Region were interested to better understand PWH's expectations and concerns of extended half‐life (EHL) factor concentrates (FC) before availability in these countries.
Journal ArticleDOI
Individualizing prophylaxis in hemophilia: a review.
TL;DR: Optimal outcomes, such as reducing or eliminating hemorrhages, preventing or minimizing joint damage, and improving quality of life, can be achieved through an individualized care approach.
References
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Journal ArticleDOI
Guidelines for the management of hemophilia.
A. Srivastava,A. K. Brewer,Evelien P. Mauser-Bunschoten,Nigel S. Key,Steve Kitchen,Adolfo Llinás,C. A. Ludlam,Johnny Mahlangu,K. Mulder,M.-C. Poon,Alison Street +10 more
TL;DR: These evidence‐based guidelines offer practical recommendations on the diagnosis and general management of hemophilia, as well as the management of complications including musculoskeletal issues, inhibitors, and transfusion‐transmitted infections.
Journal ArticleDOI
Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia.
Marilyn J. Manco-Johnson,Marilyn J. Manco-Johnson,Thomas C. Abshire,Amy D. Shapiro,Brenda Riske,Michele R. Hacker,Ray F. Kilcoyne,J. David Ingram,M L Manco-Johnson,Sharon Funk,Linda J. Jacobson,Leonard A. Valentino,W. Keith Hoots,George R. Buchanan,Donna DiMichele,Michael Recht,Deborah L Brown,Cindy A. Leissinger,Shirley Bleak,Alan R. Cohen,Prasad Mathew,Alison Matsunaga,Desiree Medeiros,Diane J. Nugent,Gregory Thomas,Alexis A. Thompson,Kevin McRedmond,J. Michael Soucie,Harlan Austin,Bruce L. Evatt +29 more
TL;DR: Prophylaxis with recombinant factor VIII can prevent joint damage and decrease the frequency of joint and other hemorrhages in young boys with severe hemophilia A.
Journal ArticleDOI
Mortality rates, life expectancy, and causes of death in people with hemophilia A or B in the United Kingdom who were not infected with HIV.
Sarah C. Darby,Sau Wan Kan,R. J. D. Spooner,Paul L. F. Giangrande,Frank Hill,Charles R. M. Hay,Christine A. Lee,Christopher A. Ludlam,Michael Williams +8 more
TL;DR: Mortality from bleeding and its consequences, and from liver diseases and Hodgkin disease, was increased, but for ischemic heart disease it was lower, and for 14 other specific causes it did not differ significantly from general population rates.
Journal ArticleDOI
A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study)
TL;DR: Prophylaxis was more effective when started early (≤36 months), with patients having fewer joint bleeds and arthropathy in children with hemophilia, particularly when it is initiated early in life.
Journal ArticleDOI
Cost of care and quality of life for patients with hemophilia complicated by inhibitors: The COCIS study group
TL;DR: Hemophilia complicated by inhibitors, a prototype of rare disease, requires high amounts of resources for management that provides a satisfactory quality of life, which is similar to that of patients with severe hemophilia without inhibitors.
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