Journal ArticleDOI
The effects of postponing prophylactic treatment on long-term outcome in patients with severe hemophilia.
Krista Fischer,Johanna G. van der Bom,Eveline P. Mauser-Bunschoten,Goris Roosendaal,Robert Prejs,Piet de Kleijn,Diederick E. Grobbee,Marijke van den Berg +7 more
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TLDR
The effect of postponing prophylaxis on long-term arthropathy was studied in a cohort of 76 patients with severe hemophilia born between 1965 and 1985, finding that most patients have their first joint bleed after the age of 2 years.About:
This article is published in Blood.The article was published on 2002-04-01. It has received 250 citations till now. The article focuses on the topics: Arthropathy & Hemarthrosis.read more
Citations
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Journal ArticleDOI
Describing the quality of life of boys with haemophilia in China: Results of a multicentre study using the CHO-KLAT.
Ling Tang,W. Xu,Changgang Li,F. Hou,Xiaoreng Feng,H. Wang,X. J. Li,W. L. Li,J. P. Liu,L. R. Sun,S. H. Wang,J. Jin,Q. Fang,K.-H. Luke,M.-C. Poon,Victor S. Blanchette,Koyo Usuba,Nancy L. Young,Runhui Wu +18 more
TL;DR: The treatment of haemophilia varies across countries and across regions within some countries, and variation has been observed in health‐related quality of life (HR‐QoL).
Journal ArticleDOI
Current and evolving features in the clinical management of haemophilia.
Antonio Coppola,Massimo Morfini,Ernesto Cimino,Antonella Tufano,Anna Maria Cerbone,Giovanni Di Minno +5 more
TL;DR: The comprehensive management of haemophilia, well beyond the treatment of bleeding, may presently focus on warranting the best possible quality of life for patients and their families, tailoring treatment choices and regimens according to specific needs.
Journal ArticleDOI
Real-life evidence in evaluating effectiveness of treatment in Haemophilia A with a recombinant FVIII concentrate: a non-interventional study in emerging countries.
Emna Gouider,S. Rauchensteiner,T. Andreeva,A. Al Zoebie,S. Mehadzic,L. Nefyodova,M. Brunn,C. Tueckmantel,Balkis Meddeb +8 more
TL;DR: This study demonstrates the effective use of rVIII‐FS in emerging countries and adds to the established safety profile of rFV III‐FS.
Journal ArticleDOI
Long-term clinical and economic outcomes in previously untreated paediatric patients with severe haemophilia A: A nationwide real-world study with 700 person-years.
K. Vepsäläinen,Pekka Riikonen,Riitta Lassila,Mikko Arola,Pasi Huttunen,Päivi M. Lähteenmäki,Merja Möttönen,Tuomas Selander,Janne Martikainen +8 more
TL;DR: The long‐term clinical outcomes and costs of treatment with high‐dose prophylaxis in PUPs from birth to adolescence, including immune tolerance induction (ITI), are evaluated.
References
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Journal ArticleDOI
Longitudinal data analysis using generalized linear models
Kung Yee Liang,Scott L. Zeger +1 more
TL;DR: In this article, an extension of generalized linear models to the analysis of longitudinal data is proposed, which gives consistent estimates of the regression parameters and of their variance under mild assumptions about the time dependence.
Journal ArticleDOI
Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B
TL;DR: It appears to be possible to prevent haemophilic arthropathy by giving effective continuous prophylaxis from an early age, and preventing the VIII:C or IX:C concentration from falling below 1% of normal.
Journal ArticleDOI
A longitudinal study of orthopaedic outcomes for severe factor‐VIII‐deficient haemophiliacs
TL;DR: It is hypothesized that no direct relationship exists between increasing factor dosage and orthopaedic outcomes over time and optimal therapeutic regimens for persons with haemophilia are established.
Journal ArticleDOI
Haemophilia prophylaxis in young patients–a long‐term follow‐up
TL;DR: To review long‐term prophylactic factor treatment in young patients with severe haemophilia A and B, focusing on the orthopaedic and radiological outcome.
Journal ArticleDOI
Primary prophylaxis in severe haemophilia should be started at an early age but can be individualized.
TL;DR: The frequency of joint bleeds and orthopaedic joint scores were evaluated in 121 patients with severe haemophilia who had started prophylactic treatment with clotting factor concentrates at least once weekly before the age of 10.
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Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia.
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