Choosing outcome assessment tools in haemophilia care and research: a multidisciplinary perspective

详细信息    查看全文

• 作者：K. Fischer ; P. Poonnoose ; A. L. Dunn ; P. Babyn ; M. J. Manco-Johnson ; J. A. David ; J. van der Net ; B. Feldman ; K. Berger ; M. Carcao ; P. de Kleijn ; M. Silva ; P. Hilliard ; A. Doria ; A. Srivastava ; V. Blanchette and On behalf of the participants of the International Symposium on Outcome Measures in Hemophilic Arthropathy
• 刊名：Haemophilia
• 出版年：2017
• 出版时间：January 2017
• 年：2017
• 卷：23
• 期：1
• 页码：11-24
• 全文大小：127K
• ISSN：1365-2516

文摘

The implementation of early long-term, regular clotting factor concentrate (CFC) replacement therapy (‘prophylaxis’) has made it possible to offer boys with haemophilia a near normal life. Many different regimens have reported favourable results, but the optimum treatment regimens have not been established and the cost of prophylaxis is very high. Both for optimizing treatment and reimbursement issues, there is a need to provide objective evidence of both short- and long-term results and benefits of prophylactic regimens.