Valoctocogene roxaparvovec and emicizumab for hemophilia A without inhibitors: effectiveness and value : final report
- Collection:
- Health Policy and Services Research
- Author(s):
- Rind, David M., author Walton, Surrey M., author Agboola, Foluso, author Herron-Smith, Serina, author Quach, Danny, author Chapman, Rick M., author Pearson, Steven D., author Bradt, Pamela, author
- Contributor(s):
- Institute for Clinical and Economic Review, issuing body. New England Comparative Effectiveness Public Advisory Council, issuing body.
- Publication:
- [Boston, Massachusetts] : Institute for Clinical and Economic Review, November 20, 2020
- Language(s):
- English
- Format:
- Text
- Subject(s):
- Antibodies, Monoclonal Genetic Therapy Hemophilia A -- drug therapy United States
- Genre(s):
- Technical Report
- Abstract:
- Hemophilia A is a condition of increased tendency to bleed due to an inherited deficiency of factor VIII, which disrupts the clotting cascade. Hemophilia A has X-linked recessive inheritance, and so predominately affects males. It is the most common of the hemophilias with an incidence of one in 5,000 male births. Patients with hemophilia A, particularly those with severe disease, are at risk for life-threatening bleeding, including intracranial bleeding, but bleeding into a joint (hemarthrosis) or muscle is more common and can lead to substantial disability. Hemarthroses cause ongoing joint inflammation and damage and also increase the likelihood of further bleeding into the same joint. To reduce the risk of bleeding, patients with severe hemophilia A have typically administered factor VIII concentrate intravenously multiple times per week. The use of factor concentrates both as treatment and prophylaxis has dramatically altered the management and clinical course of patients with hemophilia A. However, prophylaxis with factor replacement is burdensome and does not maintain patients at normal levels of factor VIII. A number of factor VIII preparations are available for prophylaxis, some with modifications to extend the half-life of the therapy, some prepared from human plasma, and some prepared using recombinant technology. Patients can develop inhibitors to factor VIII, but such patients are not considered in this report.
- Copyright:
- Reproduced with permission of the copyright holder. Further use of the material is subject to CC BY license. (More information)
- Extent:
- 1 online resource (1 PDF file (various pagings))
- Illustrations:
- Illustrations
- NLM Unique ID:
- 9918401086706676 (See catalog record)
- Permanent Link:
- http://resource.nlm.nih.gov/9918401086706676