Haemophilia prophylaxis in young patients–a long‐term follow‐up
TLDR
To review long‐term prophylactic factor treatment in young patients with severe haemophilia A and B, focusing on the orthopaedic and radiological outcome.Abstract:
Objectives. To review long-term prophylactic factor treatment in young patients with severe haemophilia A and B, focusing on the orthopaedic and radiological outcome.
Design. We received 34 patients with severe haemophilia A (n=29) and B (n=5), aged 7–22 years. Age at start of treatment was 1–4.5 years. Dosages of factor concentrate (F VIII and F IX, respectively) were 25–40 IU/kg body weight, three times a week for haemophilia A and twice a week for haemophilia B. The patients had been checked annually over a 5-year period (1990–95). Orthopaedic and radiological joint scores were evaluated according to recommendations by the World Federation of Haemophilia.
Setting. All results were obtained at the Department for Coagulation Disorders, University of Lund, Malmo University Hospital, Malmo, Sweden.
Results. Orthopaedic and radiological joint scores were found to have remained unchanged during follow-up in almost all patients and to be still zero (i.e. no unaffected joints) in 79% (n=27) of the patients.
Conclusion. There is a growing international consensus haemophilic arthropathy can be prevented by administering early high-dose prophylaxis. The results of the present investigation strongly support this opinion.read more
Citations
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Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
Catherine S. Manno,Glenn F. Pierce,Valder R. Arruda,Bertil Glader,Margaret V. Ragni,Rasko Jj,Margareth C. Ozelo,Keith Hoots,Blatt P,Barbara A. Konkle,Michael D. Dake,Robin D. Kaye,Mahmood K. Razavi,A Zajko,James L. Zehnder,Pradip Rustagi,Hiroyuki Nakai,A Chew,Debra G.B. Leonard,Debra G.B. Leonard,J F Wright,Ruth Lessard,Jurg M. Sommer,Michael Tigges,Denise E. Sabatino,A Luk,Haiyan Jiang,Federico Mingozzi,Linda B. Couto,Hildegund C.J. Ertl,Katherine A. High,Katherine A. High,Mark A. Kay +32 more
TL;DR: In this article, a single portal vein infusion of a recombinant adeno-associated viral vector (rAAV) expressing canine Factor IX (F.IX) resulted in long-term expression of therapeutic levels of F.IX in dogs with severe hemophilia B.
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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.
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TL;DR: Evidence of gene expression at low doses of vector suggests that dose calculations based on animal data may have overestimated the amount of vector required to achieve therapeutic levels in humans, and that the approach offers the possibility of converting severe haemophilia B to a milder form of the disease.
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Therapeutic genome editing: prospects and challenges
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Therapeutic genome editing: prospects and challenges
TL;DR: Current progress toward developing programmable nuclease–based therapies as well as future prospects and challenges are discussed.
References
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Journal ArticleDOI
A radiologic classification of hemophilic arthropathy.
TL;DR: A new, exclusively radiologic classification of hemophilic arthropathy is based on a review of 54 patients who had had repeated radiologic examinations of all great joints before the institution of any specific treatment.
Book ChapterDOI
Our experience in Sweden with prophylaxis on haemophilia.
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Patterns of bleeding in adolescents with severe haemophilia A.
TL;DR: The pattern of bleeding during adolescence suggests that concepts of management of arm bleeding need modifying and the overall results reflect the fact that special schools now see only the severest cases of haemophilia.
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Anders Widell,Bengt-Göran Hansson,Erik Berntorp,Torkil Moestrup,Hugo Johansson,Holger Hansson,Erik Nordenfelt +6 more
TL;DR: Intravenous transmission of HCV thus seemed highly efficient whereas sexual transmission was much less efficient.
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