Treatment of Hemophilia — More Amazing Progress

With a prevalence of 17.1 cases per 100,000 males, hemophilia A is the most frequent inherited disorder of blood coagulation. 1 After the adoption of prophylactic replacement therapy with factor VIII administered intravenously 3 or 4 times per week, patients’ life expectancy has become very close to...

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Bibliographic Details
Published inThe New England journal of medicine Vol. 383; no. 11; pp. 1068 - 1070
Main Author Mannucci, Pier M
Format Journal Article
LanguageEnglish
Published Boston Massachusetts Medical Society 10.09.2020
Subjects
Bleeding
Blood coagulation
Coagulation factors
Gene therapy
Hemophilia
Intravenous administration
Life span
Pharmacokinetics
Plasma
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Summary:With a prevalence of 17.1 cases per 100,000 males, hemophilia A is the most frequent inherited disorder of blood coagulation. 1 After the adoption of prophylactic replacement therapy with factor VIII administered intravenously 3 or 4 times per week, patients’ life expectancy has become very close to that of unaffected males. 1 Moreover, the occurrence of spontaneous bleeding and the development of musculoskeletal damage have been minimized. In addition, in the past decade, further impressive advances have occurred. 2,3 The arrival of factor VIII products with an extended plasma half-life has meant that intravenous injections that are needed to minimize bleeding can be . . .
Bibliography:SourceType-Other Sources-1
content type line 63
ObjectType-Editorial-2
ObjectType-Commentary-1
ISSN:0028-4793
1533-4406
DOI:10.1056/NEJMe2024545